Final Program - American Society of Gene & Cell Therapy

ASGCT Officers and Board of Directors 

President 

Xandra O. Breakeield, PhD 

President-Elect 

David W. Russell, MD, PhD 

Vice President 

Harry L. Malech, MD 

Secretary 

John J. Rossi, PhD 

Treasurer 

David V. Schaffer, PhD 

Board Members 

Chiara Bonini, MD 

Barry J. Byrne, MD, PhD 

Fabio Candotti, MD 

Roberto Cattaneo, PhD 

Marina Cavazzana-Calvo, MD, PhD 

Jeffrey Chamberlain, PhD 

Brendan Lee, MD, PhD 

John J. Nemunaitis, MD 

Xiao Xiao, PhD 

Ex-Oficio Board Members 

Malcolm K. Brenner, MD, PhD 

Katherine P. Ponder, MD 

Table of Contents 

A Welcome from Xandra O. Breakeield, PhD ................2 

Annual Meeting Exhibitors ....................................................3 

Annual Meeting Supporters ..................................................4 

Committee Meeting Schedule and Rosters ....................6 

Abstract Planning Committee & Reviewers ................ 12 

Excellence in Research Awards 

& Travel Grant Recipients .................................................. 14 

Award Recipients .................................................................... 15 

Faculty List ................................................................................ 16 

General Meeting Information ............................................ 22 

Hotel Information and Area Map ..................................... 26 

Convention Center Plans...................................................... 27 

Schedule-at-a-Glance ............................................................. 30 

Program Schedule ................................................................... 31 

Exhibit Hall Floor Plan .......................................................... 78 

Exhibitor Descriptions .......................................................... 79 

Exhibitor Information ........................................................... 86 

ASGCT Executive Office 

555 East Wells Street, Suite 1100 

Milwaukee, Wisconsin 53202 

Phone: 414.278.1341 

Fax: 414.276.3349 

Email: info@asgct.org 

Website: www.asgct.org 

Abstract Directory .................................................................. 87 

Abstract Author Index ........................................................ 139 

Mary Dean, JD, CAE 

Executive Director 

Ken Janowski 

Education & Meetings Manager 

Shaun Kramer 

Marketing & Outreach Manager 

Tara Withington, CAE 

Consulting Partner 

David Wood 

Senior Program Manager 

Rebecca Zimmer 

Membership & Administrative Coordinator 

Final Program SALT LAKE CITY, UTAH May 15–18, 2013 

1

A Welcome from Xandra O. Breakefield, PhD 

Dear Colleagues, 

Welcome to the American Society of Gene & Cell Therapy (ASGCT) 16th Annual 

Meeting! Salt Lake City’s rich history in the study of genetics and genealogy 

makes it a perfect city to feature this year’s latest advancements in our vibrant 

ield. The environs are also rich in majestic state parks and I hope you will 

take advantage of the chance to visit them, including Antelope Island State 

Park, as well as a guided tour of Temple Square on May 14 as part of an ASGCT 

networking adventure series. 

The program this year relects the increasing focus on reaching the 

commercialization phase of gene and cell therapeutics. On Wednesday evening, 

the Outreach Committee and BioIndustry Liaison Committee developed a two 

hour workshop on commercialization to discuss the low of funding in pipeline 

development along with some of the key conditions that must be in place in 

order to insure appropriate and eficient utilization of resources. On Saturday, 

the Translational Science & Product Development Committee will explore the 

unique issues we face in manufacturing biologics. Lastly, the BioIndustry Liaison 

Committee has organized a symposium on Friday comprising a series of talks 

and panel discussion aimed towards the latter steps of commercialization. 

In addition, we will continue to focus on our historic strengths in novel 

technologies in delivery platforms for oligonucleotide, gene and cell 

therapies, as well as advances in preclinical and clinical trials, with important 

breakthroughs on many fronts. I hope you will be able to stay for the full four 

days of programming, including the series of evening symposia sponsored by 

our foundation partners – the Foundation Fighting Blindness, the Muscular 

Dystrophy Association, the French Muscular Dystrophy Association, and the 

Alliance for Cancer Gene Therapy. 

My special thanks to all the ASGCT members, exhibitors, sponsors and attendees 

who are making this such an exciting meeting! 

Sincerely, 


President, ASGCT 

2 

Final Program SALT LAKE CITY, UTAH May 15–18, 2013

Annual Meeting Exhibitors – as of April 2013 

The American Society of Gene & Cell Therapy would like to acknowledge and thank the following exhibitors for their 

participation at the ASGCT 16 th Annual Meeting. ASGCT greatly appreciates their support and encourages all attendees to 

make time each day to visit each of these exhibitors in Hall C/D. 

Aldevron 

AltheaDx 

American Society of Gene & Cell Therapy 

Biomiga, Inc. 

BTX/Harvard Apparatus 

CellGenix GmbH 

Charles River 

Children’s Hospital of Philadelphia 

Cincinnati Children’s Hospital Medical Center 

First Biotech Inc. 

Florida Biologix 

GE Healthcare 

GenScript USA Inc 

HypOxygen 

Lovelace Respiratory Research Institute 

Mary Ann Liebert, Inc. 

Miltenyi Biotec 

MPI Research 

NanoSight 

National Gene Vector Biorepository (NGVB) 

Nature Publishing Group 

Nature Technology Corporation 

Nexcelom Bioscience 

NHLBI Gene Therapy Resource Program 

NIH Ofice of Biotechnology Activities 

Panasonic Healthcare Company of North America 

Penn Vector Core 

PerkinElmer 

PolyPlus-Transfection 

REGENX Biosciences, LLC 

SAFC 

St. Jude Children’s Research Hospital 

The Baker Company 

TriLink BioTechnologies, Inc. 

UAB Vector Production Facility 

UNC Gene Therapy Vector Core 

University of Iowa/Gene Transfer Vector Core 

University of Utah Blood & Marrow Transplant Program – 

Cell Therapy & Regenerative Medicine Facility 

VGXI, Inc. 

Waisman Biomanufacturing 

Wilson Wolf Corporation 

Thank you 16 th Annual Meeting Exhibitors! 


3

Annual Meeting Supporters – as of April 2013 

The American Society of Gene & Cell Therapy is honored to acknowledge the following organizations for their support of the 

16th Annual Meeting: 

Partner Level Support 

Printing of the Abstract Supplement 

Registration Bags 

Contributor Level Support 

Foundation Symposium 


Patron Level Support 

Conference Grant Support 


Excellence in Research Award (1) 


Outstanding New Investigator Award (1) 

Travel Grant (5) 

Excellence in Research Awards (2) 








4 


Annual Meeting Supporters 

Patron Level Support, continued 




Supported in part by March of Dimes 

Travel Coffee Mugs Travel Grant (1) 

Foundation Grant No. 4-FY13-234 




General Meeting Support 



Travel Grants (10) 



Shenzhen SiBono Gene Tech 


Travel Grant (1) Travel Grants (5) 

National Institutes of Health Grant Support 

This conference was supported by Grant Number 1R13NS083180-01 

from the National Institute of Neurological Disorders and Stroke. 

The content is solely the responsibility of the authors and does not 

necessarily represent the oficial views of the National Institute of 

Neurological Disorders and Stroke or the National Institutes of Health. 


5

Committee Meeting Schedule – as of April 2013 

Wednesday – May 15 

12:00 pm – 1:15 pm Advisory Council 250D 

12:00 pm – 1:15 pm Translational Science & Product Development Committee 250E 

12:00 pm – 1:15 pm Host Committee 251A 

12:00 pm – 1:15 pm Membership Committee 251B 

7:30 pm – 9:00 pm Molecular Therapy Editorial Board Meeting Room: Canyon BC (Hilton 

Salt Lake City Center) 

Thursday – May 16 

12:30 pm – 2:00 pm Publications Committee 250D 

12:30 pm – 2:00 pm Education Committee 250E 

12:30 pm – 2:00 pm Exhibitor Relations Committee 251A 

12:30 pm – 2:00 pm Clinical Trials & Regulatory Affairs Committee 251E 

8:00 pm – 9:30 pm Molecular Therapy: Nucleic Acids Editorial Board Meeting Room: Salon 3 (Hilton 

Salt Lake City Center) 

Friday – May 17 

11:45 am – 1:00 pm Cancer Gene & Cell Therapy Committee 250D 

11:45 am – 1:00 pm Development Committee 250E 

11:45 am – 1:00 pm Bio-Industry Liaison Committee 251A 

11:45 am – 1:00 pm New Investigator Committee 251B 

11:45 am – 1:00 pm Neurologic & Ophthalmic Gene & Cell Therapy Committee 251D 

11:45 am – 1:00 pm Outreach Committee 251E 

Saturday – May 18 

7:30 am – 8:00 am ASGCT Annual Business Meeting 150ABC 

12:15 pm – 1:45 pm 

Scientiic Committee Meetings: 

Cardiovascular Gene & Cell Therapy; 

Chemical Gene & Cell Therapy; 

Gene & Cell Therapy of Genetic and Metabolic Diseases; 

Hematologic & Immunologic Gene & Cell Therapy; 

Immune Responses to Gene & Cell Therapy; 

Infectious Diseases & Vaccines; 

Musculo-Skeletal Gene & Cell Therapy; 

Oligonucleotide and RNAi Therapeutics; 

Physical Gene & Cell Therapy & Vectorology; 

Respiratory Tract Gene & Cell Therapy; 

Stem Cell; 

Viral Gene Transfer Vectors Committee 

255EF 

12:15 pm – 1:45 pm Board of Directors Meeting 250D 

Society Annual Business Meeting 

The ASGCT Annual Business Meeting will be conducted on Saturday, May 18, 7:30 am – 8:00 am. A continental breakfast will 

be served at 7:15 am in room 150ABC of the Convention Center. 

All ASGCT Members are welcome to attend. Election results will be announced, new oficers will be installed, and the 

Society’s inances and membership statistics will be reviewed. We welcome your input and participation! 

6 


Committee Rosters 

Advisory Council 

Chair 

Terence R. Flotte, MD (2013 - 2014) 

Katherine P. Ponder, MD (2011 - 2013) 

Members 

David M. Bodine, PhD (2009 - 2014) 

Xandra O. Breakeield, PhD (2013 - 2018) 

Barrie J. Carter, PhD (2011 - 2016) 

Kenneth Cornetta, MD (2010 - 2015) 

Beverly L. Davidson, PhD (2012 - 2017) 


Carl H. June, MD (2010 - 2015) 

Luigi M. Naldini, MD, PhD (2008 - 2013) 

Arthur W. Nienhuis, MD (2008 - 2013) 

Katherine P. Ponder, MD (2009 - 2014) 

R. Jude Samulski, PhD (2012 - 2017) 

Bio Industry Liaison Committee 

Chair 

Estuardo Aguilar-Cordova (2012 - 2014) 

Members 

Dale G. Ando, MD (2010 - 2016) 

Joseph T. Bruder, PhD (2010 - 2013) 

Sunil Chada, PhD (2012 - 2015) 

Anthony T. Cheung, PhD (2008 - 2014) 

Olivier Danos, PhD (2013 - 2016) 

Mitchell H. Finer, PhD (2013 - 2016) 

Douglas J. Jolly, PhD (2013 - 2016) 

William F. Kaemmerer, PhD (2010 - 2016) 

David Kirn, MD (2012 - 2015) 

Karen Kozarsky, PhD (2007 - 2013) 

Timothy K. MacLachlan, PhD (2012 - 2015) 

Gabor M. Rubanyi, MD, PhD (2010 - 2013) 

Sean M. Sullivan, PhD (2010 - 2013) 

Thomas Tillett (2010 - 2013) 

Bruce Martin Wentworth, PhD (2013 - 2016) 

Cancer Gene & Cell Therapy Committee 

Chair 

Chiara Bonini, MD (2012 - 2014) 

Members 

Karen S. Aboody, MD (2011 - 2014) 

Margaret E. Black, PhD (2012 - 2013) 

Renier J. Brentjens, MD, PhD (2010 - 2013) 

Evanthia Galanis, MD, DSc (2011 - 2014) 

Stephen Gottschalk, MD (2007 - 2013) 

Paola Grandi, PhD (2011 - 2014) 

Akseli Hemminki, MD, PhD (2012 - 2015) 

Scott Lonning, DVM, PhD (2012 - 2015) 

J. Michael Mathis, PhD (2013 - 2016) 

John J. Nemunaitis, MD (2008 - 2014) 

Selvarangan Ponnazhagan, PhD (2012 - 2015) 

Barbara Savoldo, MD, PhD (2013 - 2016) 

Bakhos A. Tannous, PhD (2011 - 2014) 

Savio L.C. Woo, PhD (2010 - 2013) 

Masato Yamamoto, MD, PhD (2013 - 2016) 

Cardiovascular Gene & Cell Therapy Committee 

Chair 

David A. Dichek, MD (2012 - 2014) 

Members 

Michael P. Murphy, MD (2009 - 2015) 

Timothy O’Brien, MD, PhD (2009 - 2015) 

Michael J. Passineau, PhD (2010 - 2016) 

Lucio Pastore, MD, PhD (2013 - 2016) 

Gabor M. Rubanyi, MD, PhD (2010 - 2016) 

Matthew L. Springer, PhD (2007 - 2013) 

Ba-Bie Teng, PhD (2013 - 2016) 

Karen A. Vincent, PhD (2008 - 2014) 

Chemical Gene & Cell Therapy Committee 

Chair 

Ian MacLachlan, PhD (2011 - 2013) 

Kevin G. Rice, PhD (2013 - 2014) 

Members 

Stephen L. Hart, PhD (2011 - 2014) 

Philip L. Leopold, PhD (2012 - 2015) 


Suzie H. Pun, PhD (2007 - 2013) 

Kevin G. Rice, PhD (2010 - 2013) 

Cyrus R. Sainya, PhD (2010 - 2013) 

David B. Weiner, PhD (2010 - 2013) 

Clinical Trials and Regulatory Affairs Committee 

Chair 

Barry J. Byrne, MD, PhD (2012 - 2014) 

Members 

Dale G. Ando, MD (2012 - 2013) 

Wilson W. Bryan, MD (2012 - 2015) 

Jacqueline Corrigan-Curay, MD, JD (2008 - 2014) 

Katherine A. High, MD (2012 - 2015) 

David Kirn, MD (2012 - 2015) 

Maritza C. McIntyre, PhD (2013 - 2016) 

Peter K. Working, PhD, DABT (2010 - 2013) 

Development Committee 

Chair 


James M. Wilson, MD, PhD (2013 - 2014) 

Members 

Claudio Bordignon, MD (2012 - 2015) 

Malcolm K. Brenner, MD, PhD (2012 - 2015) 


E. Antonio Chiocca, MD, PhD (2012 - 2015) 


Ryszard Kole, PhD (2012 - 2015) 

Brendan Lee, MD, PhD (2013 - 2016) 


John J. Rossi, PhD (2012 - 2015) 



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Education Committee 

Co-Chairs 

Hans-Peter Kiem, MD, FACP (2013 - 2014) 

Sonia I. Skarlatos, PhD (2011 - 2013) 

Luk H. Vandenberghe, PhD (2012 - 2014) 

Members 

Aravind Asokan, PhD (2013 - 2016) 

Terry R. Bishop, PhD (2013 - 2016) 

Brian D. Brown, PhD (2011 - 2014) 

Nathalie Cartier-Lacave, MD (2011 - 2014) 

William F. Goins, PhD (2007 - 2013) 

Dirk Grimm, PhD (2011 - 2014) 

Richard P. Harbottle, PhD (2013 - 2016) 

Scott Q. Harper, PhD (2010 - 2013) 

Tal Kafri, MD, PhD (2007 - 2013) 

Elizabeth Kang, MD (2011 - 2014) 


Harry L. Malech, MD (2010 - 2013) 

Matthew H. Porteus, MD, PhD (2010 - 2016) 


James Yockman, PhD (2012 - 2015) 

Ethics Committee 

Chair 

Theodore Friedmann, MD (2012 - 2013) 

Members 

R. Michael Blaese, MD (2010 - 2013) 

Theodore Friedmann, MD (2008 - 2014) 

Dieter C. Gruenert, PhD (2010 - 2013) 

Nancy M.P. King, JD (2012 - 2015) 

Bruce E. Torbett, PhD, MSPH (2012 - 2015) 

Nelson A. Wivel, MD (2010 - 2016) 

Exhibitor Relations Committee 

Chair 

Larry A. Couture, PhD (2013 - 2014) 

Members 

Janet Benson, PhD, DABT (2013 - 2016) 

Linda B. Couto, PhD (2013 - 2016) 

J. David Enloe, Jr., BBA CPA (2013 - 2016) 

Clague Hodgson, PhD (2013 - 2016) 

Ian Johnston, PhD (2013 - 2016) 

Karen Kozarsky, PhD (2013 - 2016) 

Financial Affairs Committee 

Chair 


Members 


John J. Rossi, PhD (2010 - 2013) 

David W. Russell, MD, PhD (2011 - 2014) 

David V. Schaffer, PhD (2011 - 2014) 

Gene & Cell Therapy of Genetic & Metabolic 

Diseases and Diabetes Committee 

Chair 

Lawrence C. Chan, MD (2012 - 2014) 

Members 

Valder R. Arruda, MD, PhD (2012 - 2013) 

Alessandra Bifi, MD (2011 - 2014) 

Fatima Bosch, PhD (2010 - 2016) 

Nicola Brunetti-Pierri, MD (2012 - 2015) 

Randy J. Chandler, PhD, MB (2012 - 2015) 

Seng H. Cheng, PhD (2013 - 2016) 

Stephanie Cherqui, PhD (2011 - 2014) 

Andrew M. Davidoff, MD (2010 - 2016) 

Dwight Koeberl, MD, PhD (2013 - 2016) 

Federico Mingozzi, PhD (2012 - 2015) 

Philip Ng, PhD (2013 - 2016) 

Sihong Song, PhD (2011 - 2014) 

Peter M. Thule, MD (2013 - 2016) 

Charles P. Venditti, MD, PhD (2008 - 2014) 

Genetic Vaccines Committee 

Chair 

Jeffrey J. Molldrem, MD (2011 - 2013) 

Members 

Austin J. Barrett, MD (2010 - 2013) 

William J. Bowers, PhD (2012 - 2013) 

J. Keith Joung, MD, PhD (2012 - 2013) 

Sattva S. Neelapu, MD (2010 - 2013) 

Hematologic and Immunologic Gene and Cell 

Therapy Committee 

Chair 

Alessandro Aiuti, MD, PhD (2013 - 2014) 

Cynthia Dunbar, MD (2012 - 2013) 

Members 

Alessandro Aiuti, MD, PhD (2008 - 2013) 

Fabio Candotti, MD (2013 - 2016) 

Gianpietro Dotti, MD (2010 - 2013) 

John T. Gray, PhD (2011 - 2014) 

Helen E. Heslop, MD (2011 - 2014) 

Dennis D. Hickstein, MD (2013 - 2016) 

J. Keith Joung, MD, PhD (2012 - 2015) 

Punam Malik, MD, PhD (2013 - 2016) 

Paris Margaritis, DPhil (2011 - 2014) 

Derek A. Persons, MD, PhD (2007 - 2013) 

Pankaj Qasba, PhD (2013 - 2016) 

Barbara Savoldo, MD, PhD (2010 - 2013) 

Evangelia Yannaki, MD (2012 - 2015) 

8 



Host Committee 

Chair 



Members 

Janet Benson, PhD, DABT (2011 - 2013) 

James Brown, PhD (2013 – 2014) 

Xavier Danthinne, PhD (2011 - 2013) 

Giovanni Di Pasquale, PhD (2013 - 2014) 

Boro Dropulic, PhD (2013 - 2014) 

Howard J. Federoff, MD, PhD (2013 - 2014) 

David T. Harris, PhD (2011 - 2013) 

Eric Hoffman, PhD (2013 - 2014) 

Sung Wan Kim, PhD (2011 - 2013) 

Catherine McKeon, PhD (2013 - 2014) 

Paul Muhlrad, PhD (2011 - 2013) 

Immune Responses to Gene & Cell Therapy 

Chair 

Shulin Li, PhD (2012 - 2014) 

Members 

Catherine M. Bollard, MBChB, FRACP, FRCPA (2011 - 2014) 

Andrew P. Byrnes, PhD (2010 - 2016) 

Vincenzo Cerullo, PhD (2013 - 2016) 

Jonathan D. Finn, PhD (2013 - 2016) 

Roland W. Herzog, PhD (2010 - 2016) 

Song Li, MD, PhD (2012 - 2015) 

Gerald S. Lipshutz, MD (2010 - 2016) 

Pedro Lowenstein, MD, PhD (2012 - 2013) 

J. Michael Mathis, PhD (2013 - 2016) 

John D. Mountz, MD, PhD (2007 - 2013) 

Alistair Ramsay, PhD (2012 - 2015) 

Denise Sabatino, PhD (2008 - 2014) 

Renata Stripecke, PhD (2013 - 2016) 

Yiping Yang, MD, PhD (2010 - 2016) 

Xianzheng Zhou, MD, PhD (2012 - 2015) 

Infectious Diseases and Vaccines Committee 

Chair 

Margaret A. Liu, MD (2011 - 2013) 

Eric Poeschla, MD (2013 - 2014) 

Members 

William J. Bowers, PhD (2012 - 2015) 

Thomas W. Dubensky, Jr., PhD (2012 - 2015) 

Philip R. Johnson, MD (2010 - 2016) 

David Klatzmann, MD, PhD (2012 - 2015) 

Margaret A. Liu, MD (2011 - 2014) 

Jeffrey J. Molldrem, MD (2011 - 2014) 

Sattva S. Neelapu, MD (2010 - 2016) 

Eric Poeschla, MD (2010 - 2013) 

Steven Polyak, MD (2010 - 2013) 

Alain Rolland, PharmD, PhD (2012 - 2015) 


International Committee 

Chair 

Bobby Gaspar, MD, PhD (2013 - 2014) 

Thierry C. VandenDriessche, PhD (2011 - 2013) 

Members 

Alberto Auricchio, MD (2010 - 2013) 

Etiena Basner-Tschakarjan, MD, PhD (2013 - 2016) 

Christopher Baum, MD (2010 - 2013) 

John C. Bell, PhD (2012 - 2015) 

Bobby Gaspar, MD, PhD (2008 - 2013) 

Salima Hacein-Bey Abina, PharmD, PhD (2008 - 2014) 

Yasufumi Kaneda, MD, PhD (2012 - 2015) 

Kazunori Kataoka, PhD (2012 - 2015) 

Paula Y. P. Lam, PhD (2013 - 2016) 

Kyriacos A. Mitrophanous, PhD (2012 - 2015) 

Amit Nathwani, MD, PhD (2011 - 2014) 

Thierry C. VandenDriessche, PhD (2010 - 2014) 

Chae-Ok Yun, PhD (2012 - 2015) 

Membership Committee 

Chair 

Guangping Gao, PhD (2011 - 2013) 

Thomas Conlon, PhD (2013 - 2014) 

Members 

Donna Armentano, PhD (2011 - 2014) 

Alberto Auricchio, MD (2011 - 2014) 

Margaret E. Black, PhD (2010 - 2013) 

Michele P. Calos, PhD (2010 - 2013) 

Resy Cavallesco Wharton, PhD (2013 - 2016) 

Thomas Conlon, PhD (2012 - 2015) 


David Kirn, MD (2011 - 2014) 

Toya Ohashi, MD (2008 - 2014) 

Keiya Ozawa, MD, PhD (2011 - 2014) 

Ex-Oficio 

Robert M. Frederickson, PhD (2005 - 2014) 

Musculo-Skeletal Gene & Cell Therapy Committee 

Chair 

Kathryn R. Wagner, MD, PhD (2012 - 2014) 

Members 

Jeffrey Chamberlain, PhD (2010 - 2013) 

Joel R. Chamberlain, PhD (2013 - 2016) 

Charles A. Gersbach, PhD (2012 - 2015) 

Richard J. Gregory, PhD (2010 - 2013) 

Dwight Koeberl, MD, PhD (2010 - 2016) 

Gloria Matthews, DVM, PhD,DACVS (2013 - 2016) 

Jerry R. Mendell, MD (2012 - 2013) 

Alan J. Nixon, DVM, MS (2007 - 2013) 

Shin’ichi Takeda, MD, PhD (2010 - 2016) 

Bing Wang, MD, PhD (2010 - 2016) 

Xiao Xiao, PhD (2012 - 2015) 


9


Neurologic & Ophthalmic Gene & Cell Therapy 

Committee 

Chair 

Anne Messer, PhD (2012 - 2014) 

Members 

Robin R. Ali, PhD (2010 - 2016) 

Alessandra Bifi, MD (2011 - 2014) 

Thomas W. Chalberg, Jr., PhD (2011 - 2014) 

Florian Eichler, MD (2011 - 2014) 

David J. Fink, MD (2010 - 2016) 

William W. Hauswirth, PhD (2010 - 2016) 

Alfred S. Lewin, PhD (2012 - 2015) 

Jacques JB Mallet, PhD (2012 - 2015) 

Jodi L. McBride, PhD (2013 – 2016) 

Seshidhar Reddy Police, DVM, MS, PhD (2011 - 2014) 

Miguel Sena-Esteves, PhD (2012 - 2015) 

Mark H. Tuszynski, MD, PhD (2012 - 2013) 

John H. Wolfe, VMD, PhD (2010 - 2016) 

New Investigator Committee 

Chair 

Matthew D. Weitzman, PhD (2011 - 2014) 

Members 

Jennifer E. Adair, PhD (2011 - 2014) 

F. Charles Brunicardi, MD (2011 - 2014) 

Paul Gadue, PhD (2011 - 2014) 


Brian K. Kaspar, PhD (2011 - 2014) 

Maria P. Limberis, PhD (2012 - 2015) 

Casey A. Maguire, PhD (2012 - 2015) 

Eirini P. Papapetrou, MD, PhD (2011 - 2014) 

Miguel Sena-Esteves, PhD (2011 - 2014) 

Johan Skog, PhD (2012 - 2015) 

Luk H. Vandenberghe, PhD (2011 - 2014) 

Andy Wilber, PhD (2011 - 2014) 

Nominating Committee 

Chair 

Kenneth Cornetta, MD (2012 - 2014) 

Members 

Dale G. Ando, MD (2012 - 2013) 

Helen E. Heslop, MD (2012 - 2013) 


Kevin V. Morris, PhD (2012 - 2013) 

Oligonucleotide and RNAi Therapeutics Committee 

Chair 

Paloma H. Giangrande, PhD (2013 - 2014) 


Members 

C. Frank Bennett, PhD (2010 - 2016) 

Brian D. Brown, PhD (2010 - 2013) 

Paloma H. Giangrande, PhD (2012 - 2013) 

Dirk Grimm, PhD (2010 - 2013) 


Ryszard Kole, PhD (2011 - 2014) 

Matthew Levy, PhD (2011 - 2014) 

Paul B. McCray, Jr., MD (2008 - 2014) 

Nuria Morral, PhD (2012 - 2015) 


Marco S. Weinberg, PhD (2010 - 2016) 

Outreach Committee 

Chair 

Rachel Salzman, DVM (2013 - 2014) 


Members 

Timothy R. Cote, MD, MPH (2011 - 2014) 

Joseph C. Glorioso, III, PhD (2011 - 2014) 

Jane S. Lebkowski, PhD (2011 - 2014) 



Andra E. Miller, PhD (2011 - 2014) 




Physical Gene & Cell Therapy and Vectorology 

Committee 

Chair 

Richard Heller, PhD (2011 - 2013) 

R. Scott McIvor, PhD (2013 - 2014) 

Members 

Michele P. Calos, PhD (2007 - 2013) 

David A. Dean, PhD (2013 - 2016) 

Julie Gehl, MD, Dr.Med.Sci. (2013 - 2016) 

James G. Hecker, MD, PhD (2013 - 2016) 

Richard Heller, PhD (2011 - 2014) 

Dexi Liu, PhD (2013 - 2016) 

R. Scott McIvor, PhD (2009 - 2015) 

Carol H. Miao, PhD (2009 - 2015) 

Sean M. Sullivan, PhD (2010 - 2013) 

Ernst Wagner, PhD (2012 - 2015) 

10 



Program Committee 

Co-Chairs 

Daniel G. Anderson, PhD (2012 - 2013) 


Maria G. Castro, PhD (2011 - 2013) 

Gay Crooks, MD (2011 - 2013) 

R. Scott McIvor, PhD (2013 – 2015) 

John J. Nemunaitis, MD (2013 - 2015) 



Publications Committee 

Chair 

Cynthia Dunbar, MD (2013 - 2014) 

Francis C. Szoka, Jr., PhD (2011 - 2013) 

Members 



Stephen J. Russell, MD, PhD (2012 - 2015) 

Michel Sadelain, MD, PhD (2007 - 2013) 


Francis C. Szoka, Jr., PhD (2011 - 2014) 

Respiratory Tract Gene & Cell Therapy Committee 

Chair 

John F. Engelhardt, PhD (2013 - 2014) 


Members 

Mark J. Cooper, MD (2012 - 2015) 

David A. Dean, PhD (2010 - 2013) 

John F. Engelhardt, PhD (2010 - 2013) 


Uta Griesenbach, PhD (2010 - 2013) 

Dieter C. Gruenert, PhD (2013 - 2016) 

William B. Guggino, PhD (2013 - 2016) 

Jay K. Kolls, MD (2007 - 2013) 

Maria P. Limberis, PhD (2013 - 2016) 

Christian Mueller, PhD (2012 - 2015) 

David W. Parsons, PhD (2007 - 2013) 

Stem Cell Committee 

Chair 

Karen S. Aboody, MD (2012 - 2014) 

Members 

James Ellis, PhD (2009 - 2015) 

Paul Gadue, PhD (2013 - 2016) 

Jane S. Lebkowski, PhD (2010 - 2016) 

Carolyn Lutzko, PhD (2013 - 2016) 

Eirini P. Papapetrou, MD, PhD (2013 - 2016) 

Alice F. Tarantal, PhD (2009 - 2015) 

Translational Science and Product Development 

Committee 

Chair 

Boro Dropulic, PhD (2012 - 2014) 

Members 

Joy Cavagnaro, PhD, DABT, ATS (2010 - 2016) 

Douglas J. Jolly, PhD (2010 - 2016) 

Gene Liau, PhD (2012 - 2015) 

Dexi Liu, PhD (2013 - 2016) 


Phillip B. Maples, PhD (2013 - 2016) 

Maritza C. McIntyre, PhD (2012 - 2013) 

Alain Rolland, PharmD, PhD (2010 - 2016) 

Richard O. Snyder, PhD (2010 - 2016) 

Geoff P. Symonds, PhD (2012 - 2015) 

Dale VanderPutten, MBA, PhD (2010 - 2013) 

Gabor Veres, PhD (2012 - 2015) 

Liaison 

Jakob Reiser, PhD (2011 - 2014) 

Viral Gene Transfer Vectors Committee 

Chair 

John T. Gray, PhD (2012 - 2014) 

Members 

Mavis Agbandje-McKenna, PhD (2010 - 2016) 

Ian E. Alexander, MBBS, PhD (2011 - 2014) 

Jeffrey S. Bartlett, PhD (2013 - 2016) 

Haifeng Chen, PhD (2013 - 2016) 

John A. Chiorini, PhD (2010 - 2016) 

Kevin D. Foust, PhD (2012 - 2015) 


Stephen J. Russell, MD, PhD (2010 - 2016) 

Dmitry M. Shayakhmetov, PhD (2009 - 2015) 

Jackie Sheng, PhD (2011 - 2014) 

H. Trent Spencer, PhD (2012 - 2015) 

Weidong Xiao, PhD (2012 - 2015) 

To learn how to serve on an ASGCT Committee, visit the ASGCT Membership Booth! 


11

Abstract Planning Committee 

Each year this committee, which is made up of the ASGCT President, President-Elect, Abstract Review Chair, Program 

Committee Co-Chairs and two invited members, reviews abstracts that are suggested by the various categories for the 

Presidential Symposium and the Clinical Oral Abstract Session. 

Daniel G. Anderson, PhD 

Massachusetts Institute of Technology 

Cambridge, MA 

Xandra O. Breakefield, PhD 

Massachusetts General Hospital and 

Harvard Medical School 

Charlestown, MA 

Abstract Reviewers 

Abstract Committee Chair 

Kenneth Cornetta, MD 


Indiana University 

Indianapolis, IN 

Pedro Lowenstein, MD, PhD 

University of Michigan School of 

Medicine 

Ann Arbor, MI 


University of Washington 

Seattle, WA 

Michel Sadelain, MD, PhD 

Memorial Sloan-Kettering Cancer 

Center 

New York, NY 

RNA Virus Vectors 

Abstract Review Chair 

Dorothee von Laer, MD 

Reviewers 

Jennifer Adair, PhD* 

Brian D. Brown, PhD 

Cynthia Dunbar, MD 

Rik Gijsbers, PhD* 

AAV Vectors 


Aravind Asokan, PhD 

Reviewers 

Mavis Agbandje-McKenna, PhD 

Casey A. Maguire, PhD* 

Hiroyuki Nakai, MD, PhD 


Adenovirus and 

Other DNA Virus Vectors 


James M. Wilson, MD, PhD 

Reviewers 

Soumitra Roy, MD, PhD* 

Miguel Sena-Esteves, PhD 

Prem Seth, PhD* 

William S.M. Wold, PhD 

DNA Vectorology & Gene 

Targeting 


Matthew H. Porteus, MD, PhD 

Reviewers 

Matthew Hirsch, PhD* 

Carol Miao, PhD 

Shondra Miller, PhD* 

Andy Wilber, PhD 

Physical Methods of Delivery 


Kevin G. Rice, PhD 

Reviewers 

Todd Giorgio, PhD 

Richard Heller, PhD 

King F. Kwong, MD* 

Sean M. Sullivan, PhD 

Chemical and Molecular 

Conjugates 


Young Jik Kwon, PhD 

Reviewers 

Hai-Quan Mao, PhD 

Tatiana Segura, PhD 

Lonnie D. Shea, PhD 

Chun Wang, PhD 

Gene & Cell Therapy of Diabetes 

and Metabolic and Genetic 

Diseases 


Nuria Morral, PhD 

Reviewers 

Nicola Brunetti-Pierri, MD 

Stephanie Cherqui, PhD* 

Gerald S. Lipshutz, MD* 

Sihong Song, PhD* 

Cardiovascular Gene & Cell 

Therapy 


Thomas Weber, PhD 

Reviewers 

Michael A. Barry, PhD 

Dongsheng Duan, PhD 


Joseph E. Rabinowitz, PhD 

Neurologic Gene & Cell Therapy 


Nicholas Boulis, MD 

Reviewers 

Mimoun Azzouz, PhD* 

David J. Fink, MD 

Brian Kaspar, PhD 

Scott McPhee, PhD* 

Musculo-skeletal Gene & Cell 



Charles A. Gersbach, PhD 

Reviewers 

Annemieke Aartsma-Rus, PhD* 

Louise Rodino-Klapac, PhD* 

Jacques Tremblay, PhD 


Infectious Diseases and Vaccines 


Grant D. Trobridge, PhD 

Reviewers 

Dong Sung An, MD, PhD* 

K. Reed Clark, PhD 

Bruce L. Levine, PhD 

Renata Stripecke, PhD* 

12 


Abstract Reviewers 

Cancer - Immunotherapy 


Richard G. Vile, PhD 

Reviewers 

Steven M. Albelda, MD* 

Vincenzo Cerullo, PhD 

Jeffrey J. Molldrem, MD 

Michael I. Nishimura, PhD* 

Cancer – Oncolytic Viruses 


Grant McFadden, PhD 

Reviewers 

John C. Bell, PhD* 

David Kirn, MD 

Brian Lichty, PhD* 

Stephen J. Russell, MD, PhD 

Cancer - Targeted Gene & Cell 



Evanthia Galanis, MD, DSc 

Reviewers 

Karen S. Aboody, MD 

Helen E. Heslop, MD 

Noriyuki Kasahara, MD, PhD 

Janaiah Kota, PhD* 

Hematologic and Immunologic 

Gene & Cell Therapy 



Reviewers 

Marina Cavazzana-Calvo, MD, PhD 

Bobby Gaspar, MD, PhD 

Arnab Ghosh, MD, PhD* 

David A. Wilcox, PhD* 

Gene & Cell Therapy for 

Pulmonary Diseases 


Uta Griesenbach, PhD 

Reviewers 

Katherine J. Excoffon, PhD* 

Stephen L. Hart, PhD 

Jim Hu, PhD 

Maria P. Limberis, PhD 

Immunologic & Host Responses 

in Gene & Cell Therapy 


Pedro Lowenstein, MD, PhD 

Reviewers 

Renier J. Brentjens, MD, PhD 

Antonia Follenzi, MD, PhD* 

Maciej S. Lesniak, MD, MHCM, FACS 

Federico Mingozzi, PhD* 

Sensory (Ophthalmic and 

Auditory) Gene & Cell Therapy 


Jean Bennett, MD, PhD 

Reviewers 

Alberto Auricchio, MD 

Rajendra Kumar-Singh, PhD* 

Tonia Rex, PhD* 

Luk H. Vandenberghe, PhD 

Gene Regulation 


David W. Emery, PhD 

Reviewers 

Donna Armentano, PhD 

Michelle P. Calos, PhD 

Punam Malik, MD, PhD 

David M. Spencer, PhD 

Oligonucleotide & RNAi 

Therapeutics 


Paloma H. Giangrande, PhD 

Reviewers 

Scott Q. Harper, PhD 

Shawn E. Lupold, PhD* 

M. Corinna Palanca-Wessels, MD, PhD* 

Laura Sepp-Lorenzino, PhD 

Stem Cell Therapies 


Michel Sadelain, MD, PhD 

Reviewers 

Paul Gadue, PhD 

Hans-Peter Kiem, MD, FACP 

Jan A. Nolta, PhD 

Darwin J. Prockop, MD, PhD 

Cell Processing and Vector 

Production 


Adrian P. Gee, PhD 

Reviewers 

Gerhard Bauer, PhD 

Steven A. Feldman, PhD* 

Isabelle Riviere, PhD 

Sowmya Viswanathan, PhD* 

Special thanks to the abstract reviewers for their time and expertise! 

*Indicates irst time reviewer for ASGCT. 

To volunteer to review abstracts for the 17th Annual Meeting, visit the ASGCT Membership booth. 


13

Excellence in Research Awards & Travel Grant Recipients 

Excellence in Research Awards – For Students and Postdoctoral Fellows 

Luca Biasco 

San Raffaele Telethon Institute for Gene Therapy 

Comprehensive Study of Lentiviral [quot]Integrome[quot] in 

WAS Gene Therapy Patients 

Sourav Choudhury 

University of Massachusetts Medical School 

Identiication of Novel Vectors Capable of CNS Transduction 

in Adult Mice after Single Round Selection Using DNA Shufled 

AAV Capsid Library 

Drew Deniger 

University of Texas MD Anderson Cancer Center 

Clinical Implications of ROR1-Speciic T Cells That Target 

B-Cell Leukemia 

Charles Lai 

Massachusetts General Hospital 

Non-Invasive In Vivo Imaging, Biodistribution and Clearance 

Analyses of Intravenously Administered Extracellular Vesicles 

Rajiv Sharma 


In Vivo ZFN Mediated Targeting of Albumin as a Platform for 

Expression of Multiple Therapeutic Genes 

Barbara Smith 

University of Florida 

Acid alpha-Glucosidase Gene Replacement Therapy to the 

Diaphragm in Ventilator-Dependent Pompe Disease: One-Year 

Respiratory Motor Outcomes 

Travel Grants – For Students and Postdoctoral Fellows 

Erik Abels 

Kei Adachi 

Seemin Ahmed 

Mahzad Akbarpour 

Andrea Annoni 

Usanarat Anurathapan 

Claudia Arambula 

Cynthia Bartholomae 

Luca Biasco 

Aaron Black 

Kathleen Boerner 

Nicolas Boisgerault 

Florie Borel 

Francesca Bortolotti 

Allison Bradbury 

Elizabeth Brooks 

Christopher Burtner 

Leah Byrne 

Andrea Calabria 

Hua Cao 

Michael Castle 

Anna Castleton 

Soo Kyung Cho 

Szu-Ting Chou 

Sourav Choudhury 

Julie Crudele 

Drew Deniger 

Mark Doyal 

Theodore Drivas 

Brett Dufour 

F. Jason Duncan 

Frank Dyka 

Lauriel Earley 

Salim El-Amouri 

Colin Exline 

Susan Faust 

Antonio Filareto 

Eli Fine 

Richard Gabriel 

Pietro Genovese 

Evelyne Gicquel 

Melissa Goddard 

Diane Golebiowski 

Sara Gombash 

German Gornalusse 

Yue Guan 

Dwijit GuhaSarkar 

Marie Reine Haddad 

Chady Hakim 

Jingfen Han 

Jayson Hardcastle 

Hiromi 

Hayashita-Kinoh 

Kristin Heller 

Luiza Hernandez 

Mari Hirvinen 

Eloise Hudry 

Eric Irons 

Alok Joglekar 

Ami Kabadi 

Christine Kaeppel 

Shuichi Kamijima 

Kerstin Kaufmann 

Omkar Kawalekar 

Christiaan King 

Nico Lachmann 

Charles Lai 

Hongtao Li 

Li Li 

Shaoyong Li 

Nick Llewellyn 

Chantale Maltais 

Victoria McCurdy 

Tanner Miest 

Yoshiaki Miura 

Shohei Miyamoto 

Giridhar Murlidharan 

Bartholomew 

Naughton 

Sarah Nicolson 

Kyla Ortved 

David Ousterout 

Nunzia Pastore 

Pasquale Piccolo 

Karin Pike-Overzet 

Eric Pozsgai 

Jan-Michael Prill 

Marco Ranzani 

Maximillian Richter 

Fabrizio Rinaldi 

Julie Riviere 

Zhechao Ruan 

Giulia Ruozi 

Anna Sacher 

Lluis Samaranch 

Dwaipayan Sen 

Julien Senac 

Anurag Sharma 

Rajiv Sharma 

Shen Shen 

Kahori Shimizu 

Barbara Smith 

Arati Sridharan 

Merel Stok 

Rui-Jun Su 

Colin Sweeney 

Mehran Taherian 

William Thiel 

Lisa Tobin 

Shengdar Tsai 

Mallory Turner 

Niek van Til 

Hiu Man Viecelli 

Olga Villamizar 

Monica Volpin 

Hongjie Wang 

Rachael Watson 

Korashon Watts 

Cara Weismann 

Anna-Sophia 

Wiekmeijer 

Jun Xie 

Sea Young Yoon 

Roma Yumul 

The Excellence in Research Awards and Travel Awards were made possible through grant support from: 

14 


Outstanding New Investigator Awards 

The following Outstanding New Investigator Award winners were selected by the ASGCT Advisory Council based on 

merit and their contributions to the ield of gene and cell therapy. The recipients will present their work at the special 

Outstanding New Investigator Symposium on Saturday, May 18th from 8:00 am – 10:00 am. 


University of North Carolina at Chapel Hill 

A Sweet Side to AAV Biology 

Michael A. Laflamme, MD, PhD 


Cardiac Repair with Human Pluripotent Stem 

Cells 


University of Iowa 

Targeted-Image Guided RNA Therapies 

Ann M. Leen, PhD 

Baylor College of Medicine - CAGT 

T cell Therapy for Viruses and Cancer (TIGR) 

The Outstanding New Investigator Awards were made possible through grant support from: 

Distinguished Service Award 

The ASGCT Distinguished Service Award recognizes a person or group that has consistently fostered and enhanced 

the ield of genetic and cellular therapy. 

Sonia I. Skarlatos, PhD 

National Heart Lung Blood Institute 

National Institutes of Health 

Catherine McKeon, PhD 

National Institute of Diabetes and Digestive 

and Kidney Diseases 


Outstanding Achievement Award 

The Outstanding Achievement Award winner was selected 

by the ASGCT Advisory Council as an example of an ASGCT 

member who has achieved a pioneering research success 

through a lifetime of signiicant scientiic contributions to the 

ield of gene and cell therapy. 

Mark A. Kay, MD, PhD 

Stanford University School of Medicine 

Deciphering Vector and Non-coding RNA 

Biology: A New Frontier in Nucleic Acid based 

Therapies. 

Special Recognition Award 

The Special Recognition Award is given in acknowledgement 

and appreciation of an individual’s many scientiic 

contributions and dedication to not only the Society itself, 

but to the ield of gene and cell therapy. 

John J. Rossi, PhD 

Beckman Research Institute 

City of Hope 

The Distinguished Service Award, Special Recognition Award, and Outstanding Achievement Award Lecture will be presented 

on Friday, May 17 from 8:00 am – 9:00 am. 


15

Faculty List 

Annemieke Aartsma-Rus, PhD 

Leiden University Medical Center 

Leiden, Netherlands 

Karen S. Aboody, MD 

Beckman Research Institute of the City 

of Hope 

Duarte, CA 

Jennifer E. Adair, PhD 

Fred Hutchinson Cancer Research 

Center 

Seattle, WA 

Mavis Agbandje-McKenna, PhD 


Gainesville, FL 

Estuardo Aguilar-Cordova, PhD 

Advantagene, Inc. 

Auburndale, MA 

Alessandro Aiuti, MD, PhD 

San Raffaele-Telethon Institute for Gene 


Milano, Italy 

Ian E. Alexander, MBBS, PhD 

The Children’s Hospital at Westmead 

Sydney, Australia 


Massachusetts Institute of Technology 

Cambridge, MA 

Dale G. Ando, MD 

Sangamo BioSciences, Inc. 

Richmond, CA 

Khursheed Anwer, PhD 

Expression Genetics, Inc. 

Huntsville, AL 


University of North Carolina at 

Chapel Hill 

Chapel Hill, NC 


Telethon Institute of Genetics & 

Medicine 

Napoli, Italy 

You Han Bae, PhD 

University of Utah 

Salt Lake City, UT 

Andrew H. Baker, PhD 

University of Glasgow 

Glasgow, United Kingdom 

Alejandro B. Balazs, PhD 

California Institute of Technology 

Pasadena, CA 

Andrea Ballabio, MD 

Telethon Institute of Genetics and 

Medicine 

Naples, Italy 

David Baltimore, PhD 

California Institute of Technology 

Pasadena, CA 

Krystof Bankiewicz, MD, PhD 

University of California San Francisco 

San Francisco, CA 

Raymond T. Bartus, PhD 

Ceregene, Inc. 

San Diego, CA 

John C. Bell, PhD 

Ottawa Hospital Research Institute 

Ottawa, Canada 

C. Frank Bennett, PhD 

Isis Pharmaceuticals, Inc. 

Carlsbad, CA 

Janet Benson, PhD, DABT 


Albuquerque, NM 

Alessandra Biffi, MD 


Medicine 

Milano, Italy 

Brian W. Bigger, PhD 

University of Manchester 

Manchester, United Kingdom 

Fabrizia Bignami, PhD 

GlaxoSmithKline 

Brentford, United Kingdom 

Nicholas Boulis, MD 

Emory University School of Medicine 

Atlanta, GA 

Shannon E. Boye, PhD 



Xandra O. Breakefield, PhD 




Malcolm K. Brenner, MD, PhD 

Baylor College of Medicine 

Houston, TX 



Center 

New York, NY 

Charles R. Bridges, MD, ScD 

Sanger Heart and Vascular Institute 

Charlotte, NC 

Philip J. Brooks, PhD 

National Center for Advancing 

Translational Sciences (NCATS) 


Bethesda, MD 


Mount Sinai School of Medicine 

New York, NY 

Nicola Brunetti-Pierri, MD 


Medicine 

Napoli, Italy 

Ana Buj Bello, MD, PhD 

Genethon 

Evry, France 


University of Florida College of 

Medicine 




National Human Genome Research 

Institute 

Bethesda, MD 

Mario R. Capecchi, PhD 



Drew E. Carlson, PhD 


National Heart, Lung, and Blood 

Institute 

Bethesda, MD 

Dana Carroll, PhD 

University of Utah School of Medicine 


Barrie J. Carter, PhD 

BioMarin Pharmaceutical 

Seattle, WA 

16 


Faculty List 

Thomas W. Chalberg, Jr., PhD 

Avalanche Biotechnologies, Inc. 



University of Washington School of 

Medicine 

Seattle, WA 

Lawrence C. Chan, MD 


Houston, TX 

Randy J. Chandler, PhD, MB 


National Human Genome Research 

Institute 

Bethesda, MD 

Lieping Chen, MD, PhD 

Yale University of School of Medicine 

New Haven, CT 

Theresa Chen, PhD 

Food and Drug Administration 

Rockville, MD 

Jianjun Cheng, PhD 

University of Illinois at 

Urbana-Champaign 

Urbana, IL 

Stephanie Cherqui, PhD 

University of California, San Diego 

La Jolla, CA 

Anthony T. Cheung, PhD 

EnGene, Inc. 

Vancouver, Canada 

Ian Constable, AO 

Lions Eye Institute 

Nedlands, Australia 

Laurence JN Cooper, MD, PhD 

University of Texas M.D. Anderson 

Cancer Center 

Houston, TX 


Indiana University 


Ronald G. Crystal, MD 

Weill Medical College of Cornell 

University 

New York, NY 

Andrew M. Davidoff, MD 


Memphis, TN 

Michele De Luca, MD 

University of Modena and Reggio 

Emilia 

Modena, Italy 

David A. Dean, PhD 

University of Rochester 

Rochester, NY 

Vojo Deretic, PhD 

University of New Mexico School of 

Medicine 

Albuquerque, NM 

David A. Dichek, MD 


Seattle, WA 

Gianpietro Dotti, MD 


Houston, TX 

Dongsheng Duan, PhD 

University of Missouri School of 

Medicine 

Columbia, MO 

Cynthia Dunbar, MD 


National Heart Lung and Blood 

Institute 

Bethesda, MD 

Anja Ehrhardt, PhD 

Private University Witten/Herdecke 

Witten, Germany 

David W. Emery, PhD 


Seattle, WA 

Christine Esau, PhD 

Regulus Therapeutics 

Carlsbad, CA 

Katherine J. Excoffon, PhD 

Wright State University 

Dayton, OH 

Lukas Flatz, MD 

University Hospital of Lausanne 

Lausanne, Switzerland 

Leonard M. Fleck, PhD 

Michigan State University 

East Lansing, MI 

Terence R. Flotte, MD 

University of Massachusetts Medical 

School 

Worcester, MA 

Antonia Follenzi, MD, PhD 

University of Piemonte Orientale ‘A. 

Avogadro’ 

Novara, Italy 

Zoe Fonseca-Kelly, PhD 


Boston, MA 

Robert M. Frederickson, PhD 

Molecular Therapy 

Seattle, WA 

Svend O. Freytag, PhD 

Henry Ford Health System 

Detroit, MI 

Keith Gagnon, PhD 

University of Texas Southwestern 

Medical Center 

Dallas, TX 


Mayo Clinic 

Rochester, MN 

Daniel Gaudet, MD, PhD 

University of Montreal 

Montreal, Canada 

Andrew Geall, PhD 

Novartis 

Cambridge, MA 

Adrian P. Gee, PhD 


Houston, TX 

Charles A. Gersbach, PhD 

Duke University 

Durham, NC 

Hamid Ghandehari, PhD 



Arnab Ghosh, MD, PhD 


Center 

New York, NY 



Iowa City, IA 


17

Faculty List 

Harvinder Singh Gill, PhD 

Texas Tech University 

Lubbock, TX 

Joseph C. Glorioso, III, PhD 

University of Pittsburgh 

Pittsburgh, PA 

William F. Goins, PhD 

University of Pittsburgh School of 

Medicine 


Stephen Gottschalk, MD 


Houston, TX 

John T. Gray, PhD 


Memphis, TN 

Uta Griesenbach, PhD 

Imperial College Faculty of Medicine 

London, United Kingdom 

Dirk Grimm, PhD 

University of Heidelberg 

Heidelberg, Germany 

Markus Grompe, MD 

Oregon Health & Science University 

Portland, OR 

Farshid Guilak, PhD 

Duke University Medical Center 

Durham, NC 

Roger J. Hajjar, MD 


New York, NY 

Joshua Hare, MD 

University of Miami Health System 

Miami, FL 


Ohio State University & Nationwide 

Children’s Hospital 

Columbus, OH 

Richard Heller, PhD 

Old Dominion University 

Norfolk, VA 

Akseli Hemminki, MD, PhD 

University of Helsinki 

Helsinki, Finland 

Wim E. Hennink, PhD 

Utrecht Institute for Pharmaceutical 

Sciences 

Utrecht, Netherlands 

Roland W. Herzog, PhD 





Houston, TX 

Matthew Hirsch, PhD 


Chapel Hill 


Jeffrey R. Holt, Ph.D. 

Boston Children’s Hospital 


Boston, MA 

Artur Isaev, PhD 

Human Stem Cells Institute 

Moscow, Russia 

Brian Johnstone, PhD 

Oregon Health and Science University 

Portland, OR 

J. Keith Joung, MD, PhD 



Carl H. June, MD 

University of Pennsylvania 

Philadelphia, PA 

Tal Kafri, MD, PhD 


Chapel Hill 



University of California, Los Angeles 

Los Angeles, CA 

Brian K. Kaspar, PhD 

The Research Institute at Nationwide 


Columbus, OH 

Kazunori Kataoka, PhD 

University of Tokyo 

Bunkyo-Ku, Japan 



Stanford, CA 

Hans-Peter Kiem, MD, FACP 


Center 

Seattle, WA 

Sung Wan Kim, PhD 




Jennerex, Inc. 


Thomas Knotts, PhD 

Brigham Young University 

Provo, UT 

Donald B. Kohn, MD 



Peter Kolchinsky 

RA Capital 

Boston, MA 

Janaiah Kota, PhD 

Indiana University School of Medicine 


Karen Kozarsky, PhD 

ReGenX Biosciences, LLC 

Washington, DC 

H. Teresa Ku, PhD 

City of Hope 

Duarte, CA 

Rajendra Kumar-Singh, PhD 

Tufts University School of Medicine 

Boston, MA 

Young Jik Kwon, PhD 

University of California, Irvine 

Irvine, CA 

King F. Kwong, MD 


National Cancer Institute 

Bethesda, MD 

Michael A. Laflamme, MD, PhD 


Seattle, WA 

Jane S. Lebkowski, PhD 

Menlo Park, CA 

Brendan Lee, MD, PhD 


Howard Hughes Medical Institute 

Houston, TX 

18 


Faculty List 



Houston, TX 

Nick Leschly 

bluebird bio 

Cambridge, MA 




Shulin Li, PhD 

MD Anderson Cancer Center 

Houston, TX 

Gene Liau, PhD 

Pizer WRD 

Cambridge, MA 

Judy Lieberman, MD, PhD 


Boston, MA 




Gerald S. Lipshutz, MD 



Cecilia Lo, PhD 

University of Pittsburgh 


Shan Lu, MD, PhD 


School 

Worcester , MA 

Katarina Luptakova, MD 

Beth Israel Deaconess Medical Center 

Boston, MA 

Lawrence Lustig, MD 

University of California, San Francisco 


Ian MacLachlan, PhD 

Tekmira Pharmaceuticals Corp. 

Burnaby, Canada 

Casey A. Maguire, PhD 



Ram I. Mahato, PhD 

University of Tennessee Health Science 

Center 

Memphis, TN 

Harry L. Malech, MD 


National Institute of Allergy and 

Infectious Diseases 

Bethesda, MD 


Cincinnati Children’s Hospital Medical 

Center 

Cincinnati, OH 

Paris Margaritis, DPhil 



Jeffrey R. Martens, PhD 

University of Michigan 

Ann Arbor, MI 

Fulvio Mavilio, PhD 

Généthon 

Evry, France 

Jodi L. McBride, PhD 

Oregon National Primate Research 

Center 

Beaverton, OR 

Cheryl L. McDonald, MD 



Institute 

Germantown, MD 

R. Scott McIvor, PhD 

University of Minnesota 

Minneapolis, MN 

Olivia Merkel, PhD 

Wayne State University 

Detroit, MI 

Anne Messer, PhD 

David Axelrod Institute/Wadsworth 

Center 

Albany, NY 

Carol H. Miao, PhD 


Seattle, WA 

Jeffrey S. Miller, MD 



Shondra M. Miller, PhD 

Washington University 

St. Louis, MO 

Federico Mingozzi, PhD 

University Pierre Et Marie Curie-Paris 6 

Genethon 

Paris, France 

Elizabeth A. Mittendorf, MD, PhD 

The University of Texas MD Anderson 

Cancer Center 

Houston, TX 

Kevin V. Morris, PhD 

Scripps Research Institute 

La Jolla, CA 

Christian Mueller, PhD 


School 

Worcester, MA 

Thomas H. Murray, PhD 

The Hastings Center 

Garrison, NY 

Muna I. Naash, PhD 

University of Oklahoma Health Sciences 

Center 

Oklahoma City, OK 

Hiroyuki Nakai, MD, PhD 

Oregon Health & Science University 

School of Medicine 

Portland, OR 

Luigi M. Naldini, MD, PhD 

University Vita-Salute San Raffaele 

Milan, Italy 

Amit Nathwani, MD, PhD 

University College London Cancer 

Institute 

London, United Kingdom 

Yoichi Negishi, PhD 

Tokyo University of Pharmacy and Life 

Sciences 

Hachioji, Japan 


Mary Crowley Cancer Research Centers 

Dallas, TX 

Philip Ng, PhD 


Houston, TX 

Arthur W. Nienhuis, MD 


Memphis, TN 


19

Faculty List 

Alan J. Nixon, DVM, MS 

Cornell University 

Ithaca, NY 

Ryan M. O’Connell, PhD 



Maria C. Palanca-Wessels, MD, PhD 


Center 

Seattle, WA 

Eirini P. Papapetrou, MD, PhD 


Seattle, WA 

David W. Parsons, PhD 

Women’s & Children’s Hospital 

Adelaide, Australia 

Michael J. Passineau, PhD 

West Penn Allegheny Health System 


Marc Penn, MD, PhD 

Summa Health System 

Northeast Ohio Medical University 

Akron, OH 

Rita Perlingeiro, PhD 



Harold Petry, PhD 

UniQure, B.V. 

Amsterdam, Netherlands 

Eric A. Pierce, MD, PhD 

Massachusetts Eye and Ear 

Boston, MA 

Eric Poeschla, MD 

Mayo Clinic 

Rochester, MN 


Washington University School of 

Medicine 

St. Louis, MO 

David L. Porter, MD 

Hospital of the University of 

Pennsylvania 


Matthew H. Porteus, MD, PhD 


Stanford, CA 

Pankaj Qasba, PhD 

National Institute of Health 

National Heart Lung and Blood 

Institute 

Bethesda, MD 

Joseph E. Rabinowitz, PhD 

Temple University School of Medicine 


Daniel Rader, MD 

University of Pennsylvania Medical 

Center 


Nicholas Restifo, MD 


National Cancer Institute 

Bethesda, MD 

Tonia S. Rex, PhD 

Vanderbilt University 

Nashville, TN 


University of Iowa College of Pharmacy 

Iowa City, IA 

Isabelle Riviere, PhD 


Center 

New York, NY 

Marcelo N. Rivolta, MD, PhD 

The University of Shefield 

Shefield, United Kingdom 

Scott H. Robbins, PhD 

Immune Design Corp. 

Seattle, WA 

Louise Rodino-Klapac, PhD 

Research Institute at Nationwide 


Columbus, OH 

Maria-Grazia Roncarolo, MD, PhD 

San Raffaele Scientiic Institute 

Milano, Italy 

Stephen M. Rose, PhD 

Foundation Fighting Blindness 

Columbia, MD 

Steven M. Rowe, MD 

University of Alabama at Birmingham 

Birmingham, AL 



Seattle, WA 


Mayo Clinic 

Rochester, MN 

Denise Sabatino, PhD 

The Children’s Hospital of Philadelphia 


Aliasger K. Salem, PhD 


Iowa City, IA 

Robert A. Sandhaus, MD, PhD 

Alpha-1 Foundation 

Miami, FL 


University of California Berkeley 

Berkeley, CA 

Andrew M. Scharenberg, MD 

Seattle Children’s Research Institute 

Seattle, WA 

Daniel Scherman, PhD 

INSERM U1022 - CNRS UMR8151 

- ParisDescares University 

- ChimieParisTech 

Paris, France 

Manfred Schmidt, PhD 

Deutsches Krebsforschungszentrum 

Heidelberg 

National Center for Tumor Diseases 

Heidelberg, Germany 

Miguel Sena-Esteves, PhD 


School 

Worcester, MA 

Susan B. Sepelak, MS, MSM 

Social and Scientiic Systems, Inc 

Silver Spring, MD 

Prem Seth, PhD 

NorthShore University HealthSystem 

Evanston, IL 

Dmitry M. Shayakhmetov, PhD 


Seattle, WA 



National Heart, Lung, Blood Institute 

Bethesda, MD 

20 


Faculty List 

Evan Y. Snyder, MD PhD FAAP 

Sanford-Burnham Medical Research 

Institute 

La Jolla, CA 

Sihong Song, PhD 



David M. Spencer, PhD 

Bellicum Pharmaceuticals, Inc. 

Houston, TX 

H. Trent Spencer, PhD 

Emory University School of Medicine 

Atlanta, GA 

Gary K. Steinberg, MD, PhD 

Stanford School of Medicine 

Stanford, CA 

Renata Stripecke, PhD 

Hannover Medical School 

Hannover, Germany 

Stephen C. Strom, PhD 

Karolinska Institutet 

Stockholm, Sweden 

Suzannah Sundby 

Smith, Gambrell & Russell, LLP 

Washington DC 

Kenzaburo Tani, MD, PhD 

Kyushu University 

Higashi-ku, Japan 

Doris A. Taylor, PhD 

Texas Heart Institute at St. Luke’s 

Episcopal Hospital 

Houston, TX 

David H. Thompson, PhD 

Purdue University 

West Lafayette, IN 

John F. Tisdale, MD 



Institute 

Bethesda, MD 

Jacques P. Tremblay, PhD 

Centre de recherche du Centre 

hospitalier de l’Université Laval 

Sainte-Foy, Canada 

Grant D. Trobridge, PhD 

Washington State University 

Pullman, WA 

Rocky S. Tuan, PhD 

McGowan Institute for Regenerative 

Medicine 


Evan C. Unger, MD 

University of Arizona Cancer Center 

Tucson, AZ 

Ludovic Vallier, PhD 

Anne McLaren Laboratory for 

Regenerative Medicine 

Cambridge, United Kingdom 

Sander van Deventer, MD, PhD 

Forbion Capital Partners 

Naarden, Netherlands 

Luk H. Vandenberghe, PhD 


Boston, MA 

Thierry C. VandenDriessche, PhD 

University of Brussels 

Leuven, Belgium 

Juan F. Vera, MD 


Houston, TX 

Gabor Veres, PhD 

bluebird bio 

Cambridge, MA 

Richard G. Vile, PhD 

Mayo Clinic 

Rochester, MN 

Samuel C. Wadsworth, PhD 

Genzyme Corporation - a Sanoi 

Company 

Framingham, MA 

Kathryn R. Wagner, MD, PhD 

The Kennedy Krieger Institute 

The Johns Hopkins School of Medicine 

Baltimore, MD 

Claes Wahlestedt, MD, PhD 

University of Miami Miller School of 

Medicine 

Jupiter, FL 

Bing Wang, MD, PhD 

University of Pittsburgh School of 

Medicine 


Sue Washer, MBA 

Applied Genetics Technologies, 

Corporation 

Alachua, FL 

Yu-Quan Wei, MD, PhD 

State Key Laboratory of Biotherapy 

Chengdu, Peoples Republic of China 

Marco S. Weinberg, PhD 

The Scripps Research Institute 

Johannesburg, South Africa 

Andy Wilber, PhD 

Southern Illinois University School of 

Medicine 

Springield, IL 


University of Pennsylvania Perelman 

School of Medicine 


Savio L.C. Woo, PhD 


New York, NY 

J. Fraser Wright, PhD 





University of North Carolina at Chapel 

Hill 


James Yockman, PhD 



Michael Young, PhD 

Schepens Eye Research Institute 

Boston, MA 

Chae-Ok Yun, PhD 

Hanyang University 

Seoul, Korea 

Assem Ziady 

Emory University 

Atlanta, GA 


21

General Meeting Information 

AMERICAN SOCIETY OF GENE & CELL THERAPY 

Mission and Vision 

ASGCT’s mission is to advance knowledge, awareness, and 

education leading to the discovery and clinical application of 

genetic and cellular therapies to alleviate human disease. 

ASGCT’s strategic vision is to be a catalyst for bringing 

together scientists, physicians, patient advocates, and other 

stakeholders to transform the practice of medicine by 

incorporating the use of genetic and cellular therapies to 

control and cure human disease. 

Abstract Volume - Molecular Therapy 

All abstracts, except Late Abstract Submissions, accepted for 

presentation at the ASGCT 16th Annual Meeting have been 

published in the May supplement of Molecular Therapy. 

Attendees will receive one copy of the supplement along 

with their registration materials. Late Abstract Submissions 

accepted for presentation have been printed in a booklet 

that will be distributed to each attendee. In addition, the 

Late Abstract Submissions will be published in the July 2013 

online issue of Molecular Therapy. 

Admission 

Oficial name badges will be required for admission to all 

ASGCT sessions. All Annual Meeting attendees receive a 

name badge with their registration bag. Name badges should 

be worn at all times inside the convention center, as badges 

will be used to control access to sessions and activities. 

Attendees are cautioned against wearing their name badges 

while away from the convention center as badges may draw 

unwanted attention to your status as visitors to Salt Lake 

City, Utah. 

Admission by Ticket Only 

Some sessions at the Annual Meeting will require an 

additional ticket to control access and capacity. The Meetthe-Investigator 

sessions scheduled at the 16th Annual 

Meeting require tickets for admittance, which are not 

included in the regular registration fees. These sessions 

are designed to provide an opportunity for participants 

to interact with experts and discuss topics related to their 

research. Sessions will begin promptly and a lunch will be 

served during the program, which will last for approximately 

one hour. Each ticket is $25. 

Please Note: Tickets for the Meet-the-Investigator sessions 

are limited to 30 participants and will be sold on a irstcome, 

irst-serve basis. You must have a ticket to gain 

entrance to a session. Ushers will be collecting tickets at 

the door of each session. Tickets may be obtained at the 

Registration Desk. 

Boxed Lunches 

Pre-ordered boxed lunches will be available for pick-up 

at the registration desk from 12:00 pm – 12:30 pm on 

Wednesday, 12:30 pm – 1:00 pm on Thursday, 11:45 am – 

12:15 pm on Friday, and 12:15 pm – 12:45 pm on Satur day. 

All boxed lunches must have been pre-ordered. 

Business Center 

There is a Business Center located in the Salt Palace 

Convention Center on the Second Level Upper Concourse, 

near Room 252. The Business Center is open from 

8:00 am – 5:00 pm. Phone: (385) 468-2228. 

Business Meeting 

Join the Society’s leadership for a business meeting and 

continental breakfast. 

Saturday, May 18, 2013 

7:30 am – 8:00 am 

A light continental breakfast will be offered at 7:15 am 

Room 150 ABC, Level 1. 

All members are welcome. See page 6 for more details. 

Camera/Recording Policy 

It is the policy of ASGCT that no cameras are permitted in 

the meeting sessions, exhibit hall, or poster sessions, with 

the exception of the oficial ASGCT photographer assigned 

by ASGCT to capture images for use in Society archival and 

promotional materials. Please refrain from taking any photos 

in those locations. Audio and/or videotaping is strictly 

prohibited. 

Your attendance at ASGCT events implies your permission 

for images captured during these events to be used for the 

purposes of ASGCT archival and promotional material and 

publications and waives your rights for compensation or 

ownership of these images. 

Cell Phones & PDAs 

For the courtesy of all presenters and attendees, please 

ensure that cell phone and PDA ringers are turned off or 

silenced during all sessions as they may interfere with 

session audio. 

Committee Meetings 

Most standing and scientiic committees will meet during 

the Annual Meeting. See page 6 for Committee Meeting 

information. 

Concierge Desk 

Salt Lake City offers a wide variety of activities and 

restaurants to meet all tastes and budgets. A Concierge Desk 

will be located outside the Exhibit Hall of the Salt Palace 

Convention Center, staffed by the Salt Lake Convention & 

Visitors Bureau, who can help you with your city needs and 

reservations. 

Concierge Desk Hours 

Wednesday through Saturday 10:00 am – 6:00 pm 

22 



Continuing Medical Education 

The ASGCT Board of Directors decided that the Society will 

not offer CME credit for the 16th Annual Meeting due to 

rising costs. 

Disclosure 

In keeping with good practices, ASGCT requires faculty, 

planning committee and oral abstract presenters to disclose 

any relevant inancial interest or other relationship with 

the manufacturer(s) of any commercial product(s) and/or 

provider(s) of commercial services that are discussed in this 

educational activity. 

Educational Methods and Materials 

Lectures, Case Presentations, Panel Discussions, Question 

and Answer Sessions, Audio/Video Presentations, Abstracts, 

Posters 

Educational Objectives 

At the conclusion of the activity, the participant should be 

able to: 

• Provide advice to patients who inquire about the 

potential of gene and cell therapy or the availability of 

open clinical trials, based on their exposure to the current 

clinical trials in gene and cell therapies. 

• Better instruct their students in medical school and other 

health venues using the state-of-the-art basic science and 

clinical trials data presented at the meeting. 

• Use the latest advances in gene and cell therapy to 

enhance their research mission, as physician scientists 

conducting basic and clinical research. 

• Demonstrate improved regulatory compliance in 

conducting gene and cell therapy clinical trials, through 

exposure to NIH and FDA faculty during the educational 

program. 

Evaluation Method 

Evaluation by questionnaire will address program content, 

presentation, and possible bias. 

Needs 

Clinical gene transfer has become increasingly complex 

due to ongoing developments in the ields of gene and cell 

therapy itself, together with bioethics, research integrity, and 

inancial conlicts, as well as federal mandates, regulations 

and guidelines. Oligonucleotide Therapies, Novel Vector 

Development, Host-Vector Interactions and Vaccine 

Therapies will be discussed as well as many other scientiic 

topics. This meeting will provide an educational forum for 

scientists and clinicians to expand their knowledge about the 

broad developments in these ields. 

Target Audience 

The target audience includes basic science and translational 

researchers, clinical investigators, physicians, postdoctoral 

fellows, graduate students, employees of federal government 

and regulatory agencies, and other healthcare professionals 

with an interest in the latest advancements in the ield of 

gene and cell therapy. 

Dates 

The ASGCT 16th Annual Meeting will begin on the morning 

of Wednesday, May 15 and continue through the evening of 

Saturday, May 18, 2013. Exhibits will be open Wednesday, 

May 15 through Friday, May 17. 

Exhibits 

Exhibits will be located in Exhibit Hall C/D of the Salt Palace 

Convention Center. The companies, organizations and 

institutions exhibiting at the ASGCT Annual Meeting provide 

the latest information on products and services directly 

related to your professional needs as well as recruit for open 

positions. An Exhibit Hall Social will be held on Wednesday 

evening and a coffee social will be offered on Thursday 

and Friday in the exhibit hall. All participants are urged to 

allow adequate time daily to visit the exhibits, as they are an 

integral part of the success of the meeting. 

Exhibit Hall Hours 

Exhibits will be open Wednesday, May 15 through Friday, 

May 17, 2013. 

Wednesday, May 15 5:15 pm – 7:15 pm 

Thursday, May 16 9:35 am – 10:30 am 

Thursday, May 16 4:00 pm – 6:00 pm 

Friday, May 17 

9:00 am – 9:45 am 


5:30 pm – 7:30 pm 

Guest Attendance 

ASGCT asks registered attendees to refrain from taking 

children, spouses, or guests to any educational session or 

functions offered at the 16th Annual Meeting. 

Internet Access & Policy 

Internet access will be available in public areas at the Salt 

Palace Convention Center to all meeting attendees. Limited 

computer stations will be provided in Exhibit Hall C/D. 

When others are waiting at the internet kiosks, please limit 

your Internet use to 15 minutes at a time so other attendees 

can access the internet as well. 

Job Bank 

At the 16th Annual Meeting, there will be a forum for 

academic institutions and companies to promote open 

positions to meeting attendees. If you have open positions, 

please bring multiple copies of your job posting to the 

meeting. Similarly, if you are seeking a job, please bring 

multiple copies of your résumé to submit. ASGCT will 

not take responsibility for résumés sent to the ASGCT 

ofice in response to a job posting. If you would like more 

information about this service, please contact Shaun Kramer 

at skramer@asgct.org. The Job exchange area will be located 

inside the Exhibit Hall. 

Lead Retrieval 

A lead retrieval system has been made available to all 

exhibitors of the ASGCT 16th Annual Meeting. Exhibitors 

may ask to scan attendee name badges with a hand held 

scanner in order to obtain attendee contact information. 


23


Location 

• The 16th Annual Meeting will be held at the Salt Palace 

Convention Center , 100 SW Temple, Salt Lake City, UT 

84101, USA 

• Exhibits and Posters will take place in Hall C/D. 

• Registration will be located on the East Registration Area 

– by the East Entrance on the 2nd loor of the Salt Palace 

Convention Center 

Materials Distribution 

Please refrain from distributing promotional materials 

throughout the meeting; these may only be distributed from 

an exhibit booth in Exhibit Hall C/D. 

No Smoking 

Smoking is prohibited at all 16th Annual Meeting sessions 

and events. 

Posters 

Abstract Posters will be on display in Exhibit Hall C/D. 

Thursday, May 16 

Abstract Poster Session I 

7:00 am – 12:30 pm Poster Setup by Authors 

12:30 pm – 6:00 pm Poster Viewing (authors present 

from 4:00 pm – 6:00 pm) 

4:00 pm – 6:00 pm Networking Reception, Exhibits, and 

Poster Session I 

6:00 pm – 6:30 pm Authors Remove Posters 


Abstract Poster Session II 

7:00 am – 11:45 am Poster Setup by Authors 

11:45 am – 7:30 pm Poster Viewing (authors present 

from 5:30 pm – 7:30 pm) 

5:30 pm – 7:30 pm Networking Reception, Exhibits, and 

Poster Session II 


Saturday, May 18 

Abstract Poster Session III 

7:00 am – 12:15 pm Poster Setup by Authors 

12:15 pm – 5:45 pm Poster Viewing (authors present 

from 3:45 pm – 5:45 pm) 

3:45 pm – 5:45 pm Poster Session III 


Call4Posters ® Pick-up Schedule 

Once again this year the ASGCT arranged with Marathon 

Multimedia to provide poster authors with the opportunity 

to create their poster online using the Call4Posters® service. 

Poster presenters who took advantage of this service may 

pick-up their pre-ordered poster at the ASGCT Registration 

Desk during the following hours. 

Tuesday, May 14 5:00 pm – 7:00 pm 

Wednesday, May 15 7:00 am – 7:30 pm 

Thursday, May 16 7:00 am – 6:30 pm 

Friday, May 17 7:30 am – 7:30 pm 

Saturday, May 18 7:30 am – 4:00 pm 

Press 

Members of the working media may register for the 16th 

Annual Meeting in the Press Room – Room 252 AB. Interview 

space, computers and internet services are available for the 

convenience of media representatives covering the meeting. 

Press must register, provide credentials, and wear their 

press badge for admittance to ASGCT sessions. Assistance 

will be provided to members of the media that would like to 

schedule interviews. 

Press Room 

The Press Room is located in the Salt Palace Convention 

Center, 252 AB. 

Press Room Hours 



Friday, May 17 5:30 pm – 7:30 pm 

Registration Desk 

Name badges, inal programs, and abstract supplements will 

be distributed at the registration desk located in the East 

Registration Area (Level 2) of the Salt Palace Convention 

Center, just inside the East Entrance. 

Registration Desk Hours 




Friday, May 17 7:30 am – 7:30 pm 


Cancellation Refund Policy 

Refund requests should have been submitted in writing to 

the ASGCT Executive Ofice prior to May 3, 2013. 

Salt Lake City Information 

Please abide by the trafic signs and lights and utilize the 

designated crosswalks at all times. See Salt Lake Fun Facts 

on the next page for more information on Salt Lake City. 

Shuttle Bus 

ASGCT will not be providing shuttle transportation for the 

16th Annual Meeting. 

24 



Speaker Ready Room 

There will be a Speaker Ready Room located in Room 250 A 

on the second level of the Salt Palace Convention Center, near 

the ASGCT Registration Desk. 

All speakers, including oral abstract presenters, must deliver 

their presentations to the Speaker Ready Room the day 

before their session or at least four hours prior to their 

presentations. Equipment is available for faculty to review 

their materials. Audiovisual personnel will be available for 

assistance. Please mark your materials (your name, session 

and speaker order) so the materials can be returned to 

you. The Society strongly encourages faculty to pre-load 

presentations in the Speaker Ready Room; those faculty who 

load presentations in the meeting rooms during the sessions 

will have that time deducted from their presentation time by 

the Chair. 

ASGCT strongly encourages faculty presenting on 

Wednesday of the Annual Meeting to check-in at the Speaker 

Ready Room on Tuesday, May 14 to avoid congestion. 

Avoid delays and check-in early! 

Speaker Ready Room Hours 





7:30 am – 7:30 pm 


Special Accessibility Needs 

If you require special accommodations under the ADA in 

order to fully participate in the meeting or the course, please 

visit the Registration Desk; ASGCT staff will be happy to 

assist you with your speciic needs. 

Salt Lake City & Utah “Fun Facts” 

• The State name Utah comes from the Native American 

Ute Tribe meaning “people of the mountains” 

• The State nickname is the Beehive State – for the 

industriousness of the Utah Pioneer 

• Utah’s state motto is “Industry” 

• On December 3, 1933 Utah became the deciding US state 

to approve the repeal of prohibition 

• There are 21 National Parks within a day’s drive 

• The original Harman’s Kentucky Fried Chicken, the irst 

KFC franchise, still operates at 3900 South State Street 

• Utah is ranked #1 for happy people 

• Utah is ranked #1 for the best hiking State in the US 

• Utah is the number one per-capita consumer of Jell-O 

brand gelatin 


25

Hotel Information and Area Map 

ASGCT 16 th Annual Meeting 

May 15-18, 2013 

1. Hilton Salt Lake City Center – HEADQUARTERS HOTEL 

2. Salt Lake Marriott Downtown at City Creek 

3. Sheraton Salt Lake City Hotel 

26 


Salt Palace Convention Center Floor Plan 


27


First Level — Exhibit Hall C/D and Meeting Rooms 

• Exhibits 

• Posters 

• Job Exchange 

• Internet Kiosks 

First Level 

• Plenary Lectures 

• Session Rooms 

(Education, Oral Abstracts, 

Scientiic Symposia) 

28 



Second Level 

• Registration 

• Speaker Ready Room 

• Meet-the-Investigators 

• Committee Meetings 

• Receptions 


29

Schedule-at-a-Glance (Program subject to change) 

Tuesday, May 14, 2013 (Pre-Meeting) 

11:00 am – 6:00 pm Salt Lake City Fun Day Activities 

4:00 pm – 6:00 pm Public Education Events 

Wednesday, May 15, 2013 

7:30 am – 8:00 am Coffee Break 

8:00 am – 10:00 am Scientiic Symposia Session I 

10:30 am – 12:00 pm Education Session I 

12:00 pm – 1:15 pm Lunch Break (Lunch Not Provided) 

1:15 pm – 2:45 pm Education Session II 

3:15 pm – 5:15 pm Oral Abstract Session I 

5:15 pm – 7:15 pm Exhibit Hall Welcome Reception 

7:30 pm – 8:30 pm New Member Welcome Reception 

7:30 pm – 9:30 pm Commercialization Workshop 

Thursday, May 16, 2013 

8:30 am – 9:35 am George Stamatoyannopoulos Lecture 

9:35 am – 10:30 am Exhibit Hall Coffee Social 

10:30 am – 12:30 pm Scientiic Symposia Session II 


Meet-the-Investigator Session & Offsite Tour 

2:00 pm – 4:00 pm Oral Abstract Session II 

4:00 pm – 6:00 pm Exhibit Hall Open: Networking Reception & Poster Session I 

6:00 pm – 7:00 pm Canadian Attendee Reception 

6:00 pm – 8:00 pm Alliance for Cancer Gene Therapy Foundation Symposium 

Muscular Dystrophy Association & Association-Francaise Contre Les Myopathies Foundation 

Symposium 

Friday, May 17, 2013 

8:00 am – 9:00 am Outstanding Achievement Award Lecture with 

Distinguished Service Award and Special Recognition Award Presentations 

9:00 am – 9:45 am Exhibit Hall Coffee Social 

9:45 am – 11:45 am Scientiic Symposia Session III 

11:45 am – 1:00 pm Lunch Break (Lunch Not Provided) 

1:00 pm – 3:00 pm Oral Abstract Session III 

3:15 pm – 5:30 pm Presidential Symposium Lecture & Top Abstracts 

5:30 pm – 7:30 pm Exhibit Hall Open: Networking Reception & Poster Session II 

7:30 pm – 9:30 pm Foundation Fighting Blindness Foundation Symposium 

International Consortium for Gene and Cell Therapy Workshop 

New Investigator Town Hall & Mentoring Reception 


7:30 am – 8:00 am ASGCT Business Meeting 

8:00 am – 10:00 am Outstanding New Investigator Symposium 

10:15 am – 12:15 pm Oral Abstract Session IV 


Meet-the-Investigator Session & Offsite Tour 

1:45 pm – 3:45 pm Scientiic Symposia Session IV 

3:45 pm – 5:45 pm Poster Session III & Reception 

5:45 pm Annual Meeting Concludes 

30 


Program Schedule, Wednesday, May 15, 2013 

Coffee Break 

7:30 am – 8:00 am 

Scientific Symposium 100 

8:00 am - 10:00 am 

ROOM: BALLROOM D 

Challenges and Success of Gene Therapy Product Approval 

CO-CHAIRS: Dale G. Ando, MD and Barry J. Byrne, MD, PhD 

SPEAKERS 

Dale G. Ando, MD 

Clinical Studies of the Infusion of ZFN CCR5 Modiied Autologous CD4 T cells (SB-728T) in HIV Subjects 

CCR5 is the second receptor for HIV in R5 trophic HIV strains and the CCR5Δ32 gene mutation confers resistance to HIV infection in 

humans. This resistance to HIV infection can be recreated in CD4 T cells by ex-vivo modiication of the CCR5 gene locus by speciic 

Zinc Finger Nucleases Clinical studies treating HIV subjects with autologous CD4 T cells modiied at CCR5 by ZFN (SB-728T) in 

order to protect against HIV have been performed. Clinical data on the pharmacology, immunologic effects and safety of these 

treatments will be presented. 


Theresa Chen, PhD 

CBER Regulatory/Preclinical Considerations for AAV Products 

The presentation will provide an overview of the current CBER perspective regarding the preclinical evaluation of AAV-based gene 

therapy products and discuss regulatory and scientiic challenges in the safety and activity assessment of these investigational 

products. According to 21 CFR 312.23 (a)(8), adequate information derived from pharmacology and toxicology studies is needed 

to support a clinical investigation that is reasonably safe and scientiically feasible. Thus, the data from preclinical studies should 

provide a better understanding of the activity, safety, vector biodistribution, and transgene expression proiles following product 

administration in appropriate animal models. These data will then be used to guide aspects of the design of early phase clinical 

trials of AAV-based products, including: 1) determination of a potentially safe starting dose level and dose escalation scheme; 2) an 

optimal route of administration/anatomic site of delivery; 3) subject eligibility criteria; and 4) an adequate clinical monitoring plan. 


Next Generation Therapies with AAV 

Evan Y. Snyder, MD PhD FAAP 

The Rationale Use of Cell-based Therapies - Viewpoint from a Scientist who Advises Regulatory Agencies 

Although it is true that regulatory agencies, when assessing applications for cell-based therapies, tend to focus primarily on issues 

of safety, they are inevitably cognizant of the rationale behind the use of particular cell type in a particular clinical context in order 

to determine whether the risk is justiied. In other words, they do pay attention to whether a presumed mechanism-of-action 

is scientiically valid and supportable by the data. Certainly the scientists and physicians who advise such regulatory agencies 

scrutinize such data. This talk will discuss both the rational and irrational use of cell therapy, particularly for the neurological 

disorders, including a number of misconceptions. 


8:00 am - 10:00 am 

ROOM: BALLROOM C 

From Blood to the Brain: Gene and Cell Therapy of Genetic Diseases and Inborn Errors of 

Metabolism 

CO-CHAIRS: Randy J. Chandler, PhD, MB and Denise Sabatino, PhD 

SPEAKERS 

Ian E. Alexander, MBBS, PhD 

Pre-Clinical Gene Therapy for Urea Cycle Disorders 

A critically important function of the liver is the detoxiication of ammonia via the urea cycle. Infants with severe urea cycle 

enzyme deiciencies commonly present early in life with life-threatening hyperammonemia, progressing to coma and death in 

the absence of medical intervention. Effective management remains challenging and necessitates liver transplantation. Ornithine 

transcarbamylase (OTC) deiciency, the most common urea cycle defect, has long been recognized as an ideal model for livertargeted 

gene therapy. This presentation will explore contemporary efforts to treat OTC and other urea cycle defects, placing these 


31



in historical context. Robust life-long phenotype correction has been achieved in adult mice after single dose treatment with AAVbased 

vectors, and the translational challenges of moving towards clinical trials involving pediatric patients are currently being 

addressed. These challenges include targeting the developing liver where hepatocellular proliferation leads to loss of episomal AAV 

vector genomes and, depending on the therapeutic context, the possible need for vector re-administration to maintain effective 

levels of transgene expression. 

Stephen C. Strom, PhD 

Placental Stem Cell Treatment of Liver Disease 

Epithelial cells isolated from human amnion (hAE) have surface marker and gene expression proiles of pluripotent stem cells. 

Our previous studies have shown that hAE mature liver genes when transplanted into the liver of mice. Accordingly hAE were 

transplanted into the liver of iMSUD –mice, a useful model of the Maple Syrup Urine Disease. While untreated animals died at 

weaning, treated animals survived and thrived on a normal protein diet out to adulthood and showed signiicant correction of 

amino acid and neurotransmitter proiles. These results suggest that hAE cells could be a useful cellular therapy for metabolic liver 

diseases. 

Alessandra Bifi, MD 

Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic 

Leukodystrophy 

Amit Nathwani, MD, PhD 

AAV Mediated Gene Transfer for Hemophilia A 

We have developed distinct approach for gene therapy of haemophilia A using adeno-associated virus (AAV) vectors which is based 

on our successful strategy for gene therapy of haemophilia B. Data from our preclinical evaluation will be presented together with 

an outline of Phase I/II trial design for Haemophilia A. 


8:00 am - 10:00 am 

ROOM: BALLROOM B 

Gene and Cell Therapies for Auditory and Olfactory Disorders 

CO-CHAIRS: Jodi L. McBride, PhD and Anne Messer, PhD 

SPEAKERS 

Jeffrey R. Holt, PhD 

Gene Therapy Strategies to Restore Sensory Function in the Inner Ear 

Sensory transduction in hair cells of the inner ear is the fundamental irst step that converts auditory information into electrical 

signals which are transmitted to the brain. Loss of sensory function in inner ear hair cells is the principle deicit in over sixty 

forms of genetic deafness. To investigate gene therapy as a strategy for restoration of auditory function our lab is focused on viral 

mediated delivery of wild-type Tmc1, mutations in which cause deafness in humans and mice. Tmc1 is expressed in hair cells and 

preliminary data indicated that AAV re-introduction of wild-type Tmc1 into hair cells of deaf mice can restore sensory transduction 

in vitro. Whether AAV-Tmc1 can restore sensory function in vivo is currently under investigation. 

Lawrence Lustig, MD 

AAV1-mediated Restoration of Hearing in the VGLUT3 Knockout Mouse 

Mice lacking the vesicular glutamate transporter-3 (VGLUT3) are congenitally deaf due to loss of glutamate release at the inner hair 

cell afferent synapse. Within two weeks of AAV1-VGLUT3 delivery, acoustic brainstem response (ABR) thresholds normalize, along 

with partial rescue of the startle response. Lastly, we demonstrate partial reversal of the morphologic changes seen within the 

afferent IHC ribbon synapse. These indings will be discussed and placed into the broader context of gene therapy for genetic forms 

of hearing loss. 

Jeffrey R. Martens, PhD 

Gene Therapeutic Rescue of an Olfactory Ciliopathy 

Olfactory dysfunction is a clinical manifestation of a growing class of human genetic diseases, termed ciliopathies. Despite 

substantial progress made in identifying genes that cause ciliopathies, therapies for these disorders are not yet available to patients. 

Here, we reported that odor detection can be restored to animals with a hypomorphic mutation in the gene encoding for the ciliary 

protein IFT88 that results in the loss of cilia on differentiated olfactory sensory neurons (OSNs). These studies represent the irst in 

vivo therapeutic treatment to reestablish cilia in a mammalian ciliopathy, and show that gene therapy is a viable curative option for 

functional rescue of the complex cilia organelle in established differentiated cells. 

32 



Marcelo N. Rivolta, MD, PhD 

Restoration of Auditory Evoked Responses by hESC-derived Otic Progenitors 

The presentation will discuss recent advances using stem cells in the search of a treatment for hearing loss. A method has been 

developed to generate ear sensory cells from human embryonic stem cells (hESCs). By activating FGF signalling on hESCs using 

ligands that induce the formation of the ear in vivo, we have generated otic progenitors that can produce sensory hair cell-like cells 

and auditory neurons. We have then taken the hESC-derived otic progenitor cells and explore if they could repair a deaf ear in a 

gerbil model of auditory neuropathy. When hESC-derived ear cells were transplanted into cochleae that have lost their auditory 

neurons the cells survived, engrafted and differentiated. Moreover, they sent projections making connections with the hair cells and 

with the brain and, more remarkably, they elicited a functional recovery represented by improved ABR thresholds. Related work, 

developing them as a platform for gene and drug discovery will also be reviewed. 


8:00 am - 10:00 am 

ROOM: 150 DEFG 

Novel Approaches to Cancer Immunotherapy 

CO-CHAIRS: Jennifer E. Adair, PhD and Elizabeth A. Mittendorf, MD, PhD 

SPEAKERS 


Elizabeth A. Mittendorf, MD, PhD 

AE37: A Novel Hybrid Vaccine for Stimulating a Helper T Cell Response 

Our group has been leading the clinical trial efforts evaluating AE37, a novel vaccine that combines AE36, a native HER2-derived 

MHC class II epitope to the Ii-Key moiety. Linkage of the Ii-key moiety increases antigen presentation in vivo leading to increased 

potency of the vaccine. Phase I trials have demonstrated AE37 to be safe and effective in eliciting a HER2-speciic immune response. 

An ongoing phase II trial has shown evidence of clinical eficacy; at the time of a planned analysis at 24 month median follow-up, 

the disease-free survival rate was 90% in vaccinated patients versus 83% in non-vaccinated controls, a 41% relative risk reduction. 

This talk will focus on the preclinical and early clinical trial data available for the AE37 vaccine. 


Clinical Update of bi-shRNA furin/GMCSF DNA Transfected Tumor Vaccine (FANG) in Cancer Patients 

Exciting the immune system to attack cancer involves 3 critical components: education of tumor antigens, enhancement of cancer 

targeting immune function, and blockade of pro-cancer immune inhibitors. FANG is a novel “triad” vaccine which utilizes both 

cellular and DNA technology to accomplish all 3 components. Speciically, autologous harvested tumor cells are transfected with a 

dual plasmid involving a GMCSF DNA cassette and a bifunctional RNA interference DNA cassette targeting furin, the proprotease 

converting enzyme of TGFß1 and TGFß2. Phase I trial involving 42 advanced cancer patients (Senzer et. al. Mol Ther 2012: 679- 

686) demonstrated patient safety, veriication of product mechanism and correlation of signiicant survival improvement with 

induction of activated T-cells as measured by ELISPOT response. Follow up results of the Phase I trial with demonstration of median 

survival advantage in comparison of FANG treated patients to FANG not treated patients of 562 days vs. 181 days (p




8:00 am - 10:00 am 

ROOM: 151 ABCG 

Novel Structural and Functional Assessment of the Respiratory Tract: Potential New Endpoints 

for Gene and Cell Therapy 

CO-CHAIRS: David A. Dean, PhD and Christian Mueller, PhD 

SPEAKERS 

David W. Parsons, PhD 

Developments in Functional X-ray Imaging: ASL, MCT and “Pinpoint Spirometry” 

Driven by a desire to rapidly, accurately and reliably measure the effect of airway gene therapy treatments for Cystic Fibrosis 

in mice, we have been developing several novel analytical options for airways using synchrotron phase contrast X-ray. The high 

lux and coherence of synchrotron X-rays underlie our ability to visualize mouse airway-surface anatomy and activity in-vivo, to 

sub-micron resolution; ultra-fast imaging of dynamic lung movement in 4-dimensions to reveal regional airlows is also enabled. 

Importantly, all of these new approaches are designed to be non-invasive and allow airway health and function in intact animals 

to be examined. Alterations in the depth of the airway surface liquid (ASL) and altered Mucociliary Transit (MCT) are the earliest 

physiological effects of CFTR activity that can produce visible change in airway surface structure and function, and are key markers 

of CF airway health to be improved by an effective gene therapy. The core methods for the image capture, analysis, and presentation 

of both ASL and MCT markers are rapidly developing to provide new endpoint tools. The application of velocimetry techniques to 

synchrotron CT data has created a powerful new tool for regional lung health assessment (”pinpoint spirometry”) that is likely to be 

of particular value in animal models, and is also well suited to translation into a clinically-applicable system. 

Cecilia Lo, PhD 

Respiratory Motile Cilia Dysfunction in Congenital Heart Disease Patients and Contribution to Worse 

Postsurgical Outcomes 

Steven M. Rowe, MD 

Functional Anatomic Imaging of the CF Airway 

A critical barrier causing a roadblock in our understanding of CF pathogenesis is the lack of tools available for visualizing the 

important microstructural, functional, and biomechanical features of the respiratory mucosa and mucus in vivo. Recently, our 

laboratories have developed an innovative technology, termed 1-µm resolution optical coherence tomography (µOCT), which 

enables real-time cross-sectional microscopy of the functional epithelial surface of living airways and can be readily combined with 

measures of ion transport in vivo. With µOCT, we have been able to simultaneously and quantitatively monitor airway surface liquid 

(ASL) and periciliary layer (PCL) depths, ciliary beating, and mucociliary transport (MCT) in situ, while also measuring mucus 

viscosity by native particle tracking techniques without the use of contrast dyes, exogenous microparticles, or other manipulations. 

Key manifestations of the underlying CF defect can readily be observed, and an in vivo probe is currently under development. In 

addition to answering important questions regarding CF pathophysiology, this technology provides a clear path towards the use of 

µOCT for monitoring functional responses to novel therapeutics. 


Development of an AAV-based Prophylactic Vaccine against Pandemic Inluenza 

We will discuss the development of an effective prophylactic vaccine against inluenza. This vaccine is based on AAV9 vector and 

expresses a broadly neutralizing antibody against inluenza that protects relevant animal models against challenge with H5N1 and 

H1N1. 

34 




8:00 am - 10:00 am 

ROOM: 150 ABC 

Polymeric Gene Delivery 

CHAIR: Sung Wan Kim, PhD 

SPEAKERS 

Wim E. Hennink, PhD 

Decationized Polyplexes: Novel Polymeric Gene Delivery System with Reduced Toxicity 

The clinical applicability of polymers as gene delivery systems depends not only on their eficiency, but also on their safety. The 

cytotoxicity of these systems remains a major issue, mainly due to their cationic nature. Therefore, it is highly preferable to have a 

system based on biocompatible neutral polymers, lacking polycations, without compromising the DNA condensing and protecting 

capacities. We introduce a concept to obtain a neutral polymeric gene delivery system, through a 3-step process to generate 

polyplexes with a core of disulide crosslinked poly(hydroxypropyl methacrylamide) (pHPMA) in which plasmid DNA (pDNA) is 

entrapped and a shell of poly(ethylene glycol) (PEG). 


Ram I. Mahato, PhD 

Emerging Trends in Combination Therapy with RNAi 

Treatment strategies relying on a single small molecule drug, or silencing of a single gene often fail, while simultaneous targeting 

of multiple cellular pathways is more likely to succeed. RNA interference (RNAi) is a promising approach for treating diabetes and 

cancer. By pooling siRNAs targeting two different regions of the same target gene, or combining two siRNAs against two different 

genes, or even co-formulating small molecule drug with speciic siRNA or miRNA, we hope to improve islet transplantation and 

treat advanced prostate and pancreatic cancer. In this presentation, I will discuss our recent results on RNAi technologies for 

treating diabetes and cancer. 

Chae-Ok Yun, PhD 

Harnessing Adenovirus for Tumor Targeting by Nanocomplex 

A challenge to develop adenovirus (Ad)-mediated therapeutics has been issued to treat metastatic cancer via systemic 

administration. For effective gene therapeutics against primary and metastatic lesions, a systemically injectable tumor-targeting 

Ad vector system must be developed. Systemic delivery of Ad, however, is hampered by immune response against Ad, short half-life 

in the circulation, liver uptake, and low accumulation at target disease sites. Modiication of the Ad surface allows Ad to circulate 

in the bloodstream for a longer time, to be not targeted to the liver, and to passively accumulate in tumor sites via enhanced 

permeation and retention effects. The addition of afinity tags results in active targeting and high eficacy. Strategies including 

addition of polymer complexes, chemical modiications, and targeting moieties for the development of systemically injectable Ad 

gene therapeutic carriers will be discussed. 

Kazunori Kataoka, PhD 

Polymeric Micellar Nanocarriers for Gene and Oligonucleotide Delivery 

Focus of my talk will be on the use of self-assembly structures from multi-functional block copolymers, particularly polymeric 

micelles, for gene and oligonucleotide delivery. Importance of peptide ligands to facilitate extravasation as well as to control 

intracellular traficking of polymeric micelles will be discussed. Also, I will present our new strategy to delivery messenger RNA to 

target organ. 


35



Education Sessions 

The goal of the ASGCT Education Program is to provide participants with a broad background and introduction to topics relevant to the 

ield of gene and cell therapy. Education Sessions will be subdivided into two headings. “Topical Review” sessions will provide a broad 

perspective of an area of importance to the gene and cell therapy community such as major vector systems, speciic target organs or 

diseases, and funding/regulatory issues. “Emerging Field Review” sessions will provide a broad perspective of a technological or scientiic 

discipline that has recently become of great signiicance to ASGCT members. 

Education Session 110 

10:30 am - 12:00 pm 


Emerging Field Review: Gene Targeting 

Gene targeting, also known as genome editing, has become an increasingly feasible gene therapy strategy. In this education, three pioneers 

in the ield will describe different approaches to gene targeting. After this session, attendees should be able to understand the different 

approaches to gene targeting and which strategy might be best for their speciic gene therapy application. 

CHAIR: Matthew H. Porteus, MD, PhD 

SPEAKERS 


AAV-Mediated Gene Targeting 

This presentation will describe the mechanism and applications of AAV-mediated gene targeting, which has been used to target 

over 100 genes in more than 50 different cell types. Sequence changes can be introduced at high frequencies, with up to 1% of 

infected cells undergoing gene targeting. Insertions, deletions and substitutions can all be accurately introduced, and additional 

unwanted mutations are not found at targeted loci. Site-speciic nucleases are not required to obtain high targeting frequencies, so 

there is no risk of introducing DNA breaks at off-target sites. 

J. Keith Joung, MD, PhD 

Engineered TALENs and CRISPR-Cas RNA-Guided Nucleases (RGNs) for Targeted Genome Editing 

Engineered site-speciic nucleases have emerged as a broadly applicable technology for eficiently inducing targeted alterations 

in the genomes of living cells and organisms. This talk will provide an introduction to two engineered nuclease platforms: 

transcription activator-like effector nucleases (TALENs) and CRISPR-Cas RNA-Guided Nucleases (RGNs). The strengths and 

limitations of each of these platforms will be discussed. In addition, publicly available methods and resources for practicing these 

technologies will be reviewed. 

Dana Carroll, PhD 

Nuclease-mediated Gene Targeting 

With new and rapidly developing technology, it is now possible to make knock-outs and knock-ins in virtually any gene in a wide 

range of cells and organisms. The secret is the development of nucleases that can be designed to cleave arbitrarily chosen genomic 

DNA sequences with high speciicity. Cellular repair processes create localized mutations at the nuclease-induced break, or copy 

novel sequences from a user-provided template. This talk will cover two classes of targetable cleavage reagents, zinc-inger 

nucleases (ZFNs) and TALENs. The research and therapeutic potential of these reagents is enormous, and ZFNs are currently in 

clinical trials. 


10:30 am - 12:00 pm 


Emerging Field Review: Ocular Gene Therapy: Tools and Genes 

Following the success of the clinical gene therapy trials for an early onset form of inherited blindness called LCA2, the momentum in the 

ield of ocular gene therapy is palpable. In this session, we try to capture this excitement by providing an in depth review into the genetics 

of vision and blindness and the technologies that are being developed to therapeutically intervene in the disease course using gene transfer. 

This educational session also will discuss disease gene discovery in the genomic era, genetic diagnosis for inherited forms of blindness, gene 

therapies for blindness under development, and AAV and nanoparticle vector systems for retinal targeting. 

36 



CHAIR: Luk H. Vandenberghe, PhD 

SPEAKERS 


Gene Discovery in Inherited Retinal Blindness 

IRDs important causes of blindness. Proof of concept clinical trials gene therapy for RPE65 LCA suggest that these disorders 

are amendable to treatment with gene therapies. The genetic heterogeneity of these disorders present challenges for genetic 

diagnostic testing. The use of next-generation sequencing techniques for genetic diagnostic testing and disease gene discovery 

research for these disorders will be discussed. 

Shannon E. Boye, PhD 

AAV Gene Transfer to the Retina 

Clinical trials for RPE65-LCA (LCA2) have demonstrated the ability to target a subretinally- delivered therapeutic transgene to 

the retinal pigment epithelium (RPE) thereby restoring retinal function and visually-evoked behavior to these patients. However, 

because most retinal degenerations are caused by mutations in photoreceptor (PR)- speciic genes, there is a great need to develop 

PR-targeted gene therapies. This presentation will review and compare known PR-targeted AAV vectors, with emphasis placed on 

results recently obtained in higher order species following subretinal delivery. The future development of intravitreal-delivered 

vectors targeted to photoreceptors will also be discussed. 


Muna I. Naash, PhD 

Nanoparticle-mediated Ocular Gene Delivery 

The eye is an organ that is well-suited for the development and testing of novel therapeutic approaches. It is easily accessible and 

allows local application of therapeutic agents with reduced risk of systemic effects. Need exists for the development of non-viral 

therapeutic approaches for ocular diseases. Our lab and others have investigated the potentials of nanotechnology for ocular 

delivery of therapeutic genes. Investigations thus far have highlighted great potentials for nanoparticles to be a successful approach 

for ocular gene delivery. Our group has focused on the eficacy of compacted DNA nanoparticles for the treatment of different 

diseases, particularly those associated with the retina and retinal pigment epithelium. We have shown that nanoparticle treatment 

leads to eficient transfection of ocular cells, long term gene expression, and exerts no toxic effects on the eye even after multiple 

injections. These nanoparticles mediate signiicant functional rescue in models of retinitis pigmentosa, Stargardt macular dystrophy 

and Leber’s congenital amaurosis. They have no limitations on the size of the genetic cargo and effective gene expression has been 

demonstrated with vectors up to 20 kb in the lung and 14 kb in the eye, making them an ideal complement to AAVs especially for 

delivery of large genes. Furthermore, in a side-by-side comparison study with AAV, we recently reported that nanoparticles can 

drive gene expression on a comparable scale and longevity to AAV. 


10:30 am - 12:00 pm 


Topical Review: A Primer on Non-Viral Delivery 

CO-CHAIRS: Ian MacLachlan, PhD and Kevin G. Rice, PhD 

SPEAKERS 


Liposomal and Lipid Nanoparticle Formulations for the Delivery of siRNA 

David H. Thompson, PhD 

Non-Viral Vectors: Correlations Between Structure and Performance 

A vast array of materials have been screened as potential non-viral vectors, including cationic lipids, hyperbranched polymers, 

dendrimers, cyclodextrin polymers, and nanoparticle carriers, however, few of the materials developed for this application have 

been systematically evaluated for their structure-property relationships. This presentation will begin by briely reviewing the 

various strategies used before focusing on those systems where it has been possible to correlate biological performance with the 

structure and/or physical properties of the non-viral vector. Finally, new directions in non-viral vector development will be covered 

with an emphasis on mechanism-based studies. 


Principles of DNA Polyplex Design for In Vitro and In Vivo Gene Delivery 


37




10:30 am - 12:00 pm 


Topical Review: Gene and Cell Therapy Approaches to Hemoglobinopathies 

This session will introduce basic, translational and clinical efforts to provide therapeutic advances for the two major Hemoglobinopathies: 

sickle-cell anemia (SCD) and beta-thalassemia’s using gene therapy approaches. Elevation of HbF production has been a long-standing 

goal of therapy for ß-thalassemia and sickle cell disease (SCD) as its expression provides a potent attenuation of many of the most severe 

complications of disease phenotype. The central theme of this session will be improving gene transfer and gene correction in human 

hematopoietic stem and progenitor cells to provide more effective and safe therapies for blood cell disorders. In addition, efforts and the 

lessons learnt from gene therapy of Severe Combined Immunodeiciency (SCID) disease. 

CO-CHAIRS: Pankaj Qasba, PhD and Sonia I. Skarlatos, PhD 

SPEAKERS 


Gene and Cell Therapy Approaches to Hemoglobinopathies 

Dr. Malik will present an overview of genetic therapies for hemoglobiniopathies, a review of the literature, the current state of the 

ield and future promising ways of gene editing and reprogramming. 

John F. Tisdale, MD 

Stem Cells as Gene Therapy Vehicles for the Treatment of Disorders of Globin Synthesis 

Sickle cell disease (SCD) is a recessive genetic disease that results from the production of an abnormal hemoglobin that is prone 

to polymerization upon deoxygenation causing severe morbidity and early mortality. Although palliative therapies have been 

developed, conventional treatment remains unsatisfactory. Signiicant advances in cellular and molecular biology have enabled 

the development of a mounting armamentarium of tools to allow correction at the hematopoietic stem cell (HSC) level. Allogeneic 

HSC transplantation, a form of genetic therapy, is an established curative approach through replacement of the defective organ 

with that of a donor carrying the normal genotype; however, procedural toxicities limit this approach to only severely affected 

children. Nonmyeloablative approaches based upon basic and preclinical studies have now allowed for the successful application 

of this approach in adults with severe disease, with disease reversion in the majority of patients treated through the attainment of 

stable mixed hematopoietic chimerism. Nonetheless, this approach remains limited by donor availability, and the development of 

gene transfer directed at autologous HSCs remains a desirable alternative. Approaches using autologous transplantation of HSCs 

modiied by lentiviral vectors have demonstrated expression of the human beta-globin protein at clinically signiicant levels, and 

this approach is now being developed for clinical application in SCD. The progress toward the goal of these HSC based therapies will 

be reviewed. 


Lessons Learned from Gene Therapy for SCID 

The implementation and performance of clinical trials of gene therapy using hematopoietic stem cells is a complex process. After 

proof-of-principle studies demonstrate potential disease modifying activity, preclinical activity and toxicology data need to be 

generated, along with clinical protocol, consent documents, etc. to support regulatory applications. Subjects are treated in the 

high acuity setting of hematopoietic stem cell transplantation (HSCT) which may include the use of myeloreductive conditioning, 

although no immune suppression pre- or post-transplant is needed, due to the use of autologous cells. Ultimately, clinical 

acceptance of gene therapy as a standard of care will depend on demonstrating equivalent or greater eficacy and improved 

outcomes or reduced costs, compared to allogeneic HSCT. Experience with gene therapy for severe combined immune deiciency 

may inform efforts to develop similar approaches to hemoglobinopathies; persistence and patience are needed. 


10:30 am - 12:00 pm 


Topical Review: Vector Design 

CO-CHAIRS: Alberto Auricchio, MD and Dirk Grimm, PhD 

38 



SPEAKERS 

Anja Ehrhardt, PhD 

Viral Hybrid-Vectors for Stable Tranduction of Mammalian Cells: Molecular Design, Eficacy and Applications 

Viral hybrid-vectors combine the high transduction eficiencies of recombinant viruses with improved genetic elements for 

stabilized transgene expression. This tutorial provides a state-of-the-art overview of available viral hybrid-vector systems for 

random somatic integration (Sleeping Beauty transposase), targeted integration (designer nucleases) and episomal persistence 

of the transgene (plasmid replicons). Advantages but also limitations of each vector system will be discussed and safety issues 

addressed. Furthermore, recent in vivo applications will be mentioned. 


AAV - From the Genome to the Capsid 

Vectors based on the adeno-associated virus (AAV) are being tested in humans and the initial safety and eficacy results in several 

tissues, including liver, muscle, retina and central nervous system are extremely encouraging. The availability of hundreds of 

naturally-occurring AAV serotypes, as well as of mutagenized variants with improved and different transduction proiles provide 

a theoretical endless source of vectors for in vivo gene therapy. Sophisticated strategies were developed to tailor transgene 

expression to speciic cell types within the AAV cargo capacity. In addition, efforts are being made to expand AAV cargo capacity 

for the expression of large transgenes. Improvements on both the outside (capsid) and inside (genome) of AAV vectors will be 

discussed in the context of the retina which is an excellent target tissue to challenge the versatile AAV platform. 


Philip Ng, PhD 

Helper-dependent Adenovirus: Design, Production and Applications 

This education session will discuss optimal design, eficient production and novel applications of helper dependent adenovirus for 

gene and cell therapy. 

Lunch Break (On Own - Not Provided; Pre-ordered boxed lunches available for pick up at Registration Counter) 

12:00 pm - 1:15 pm 


1:15 pm - 2:45 pm 


Emerging Field Review: Therapeutic Antibody Gene Delivery 

Recent advances in monoclonal antibody discovery, isolation, and expression have created unprecedented opportunities to utilize this class 

of proteins for broad therapeutic beneit. A number of studies have highlighted several potential applications for the use of antibodies 

in vivo, ranging from the prevention of infectious diseases, to the treatment of drug addiction. This session will focus on several of these 

applications and will highlight strategies aimed at maximizing vector-based expression of antibodies in vivo. 

CO-CHAIRS: Alejandro B. Balazs, PhD and James M. Wilson, MD, PhD 

SPEAKERS 


Inluenza Antibodies and Airway Delivery 


Gene Transfer Strategies to Treat Addiction 

Addiction to drugs is a major worldwide problem for which there is no effective therapy. Using cocaine and nicotine as examples 

of commonly used addictive drugs, we have developed two platform strategies to evoke high-titer antibodies speciic for the antiaddictive 

drug. In the irst strategy, based on the highly immunogenic nature of the adenovirus capsid, analogs of the addictive 

drug are bound to adenovirus capsid proteins to create active vaccines that generate high-titer anti-drug antibodies. In the second 

strategy, the gene coding sequences for monoclonals against the addictive drug are expressed by adeno-associated virus gene 

transefer vectors capable of generating, with a single administration, persistent high titers of anti-drug antibodies. In experimental 

animal models, both strategies are highly effective, providing new paradigms for anti-addictive molecular vaccines. 

Alejandro B. Balazs, PhD 

Expressing Antibodies in vivo via Adeno Associated Virus Vector for Vectored ImmunoProphylaxis 

Recent advances in vector-based antibody delivery have created the potential for engineering humoral immunity to address 

problems ranging from infectious disease to drug addiction. These efforts are often hampered by dificulties in achieving 

therapeutic levels of antibody expression. This presentation will focus on means of improving expression of antibodies in vivo and 

summarize the challenges of working with this novel application of gene transfer. 


39




1:15 pm - 2:45 pm 


Topical Review: Cancer Gene Therapy 

The ield of Cancer Gene Therapy has seen many recent developments concerning applications to treat different tumors using a variety 

of novel vector systems that are now being exploited in human clinical trials. This session will deal with pre-clinical accomplishments 

concerning the use of chimeric antigen receptor (CAR) modiied T-cells directed against tumor associated antigens, the use of novel tumorselective 

replication-competent retroviruses expressing prodrugs, and the use of bifunctional shRNAi nanoplex technology for advanced 

methods of killing tumors using gene/cell therapies. Recent signiicant progress in clinical trials resulting from these approaches will also be 

presented. 

CHAIR: William F. Goins, PhD 

SPEAKERS 


Clinical Update of bifunctional (bi)-shRNAi Nanoplex Technology in Cancer 

Several RNAi approaches (miRNA, siRNA, shRNA, bi-shRNA) are being explored to exquisitely disrupt post transcriptional gene 

silencing of cancer relevant signals. Twenty-six clinical studies involving RNAi technology have been published. bi-shRNAi (Issued 

Patent No. 8,252,526) utilizes a dual mechanism to knockdown sequence speciic targets by engaging both siRNA and miRNA-like 

RNA-inducing silence complex. The bi-shRNAi technology was initially introduced into the clinical areas to target a proprotease 

converting enzyme, furin, as one of 3 components involving a novel FANG vaccine (Senzer et. Al. Mol Ther 2012: 679-686) in which 

clinical demonstration of protein knockdown to the designed target, furin and downstream signals (TGFß1, ß2) was greater than 

90%, and immune response in correlation with survival advantage was demonstrated. In an effort to expand bi-shRNAi technology 

and to target other potentially therapeutic molecular signals, we have utilized a nanoplex approach by incorporating the bi-shRNAi 

plasmid into a bilamellar invaginated vesicle (BIV) (Issued Patent No. 7,037,520) which can be decorated with reversible masking 

lipids and targeting small molecule ligands (Issued U.S. Patient No. 8,333,988). Results of initial preclinical and clinical studies will 

be reviewed. 


Development of Novel Vectors and Strategies for Gene Therapy of Cancer 

Oncolytic virotherapy, engineered T cells and cancer vaccines, and stem cell-based tumor-targeting strategies are currently 

being evaluated in the clinic and show considerable promise. Newer approaches seek to further enhance the potency of RNA 

oligonucleotide-based therapeutics, replicating virus vectors, and engineered cell therapies, while maintaining tumor-selectivity 

and safety. An overview of recent advances showing signiicant promise for the development of clinically effective gene therapy 

against cancer will be presented. 

Gianpietro Dotti, MD 

Chimeric Antigen Receptor (CAR) Engineered T-cell Cancer Therapies 


1:15 pm - 2:45 pm 


Topical Review: Gene Therapy to the Marketplace 

CHAIR: H. Trent Spencer, PhD 

SPEAKERS 

H. Trent Spencer, PhD 

Replacing Treatments with Cures: Potential Therapeutics for Hemophilia A 

Hemophilia A is a monogenic bleeding disorder currently treated by protein replacement therapy. Gene therapy provides an 

alternative treatment that can eliminate the need for recombinant protein administration. Preclinical and clinical data show a long 

lasting/lifelong treatment is feasible, and various models have shown gene therapy for hemophilia A is also inancially attractive, 

which further justiies the development of these curative treatments. This presentation will summarize hemophilia treatment 

options with respect to their market potentials. 

R. Scott McIvor, PhD 

Discovery Genomics: Development of the Sleeping Beauty Transposon System for Gene Therapy 

40 



Khursheed Anwer, PhD 

IL-12 Gene Therapy: From Bench to Clinic 

This presentation describes the clinical and preclinical development of EGEN-001, an interleukin-12 (IL-12) gene expression 

plasmid formulated with a biocompatible delivery lipopolymer, PEG-PEI-Cholesterol (PPC), for the treatment of peritoneally 

metastasized gynecological and gastrointestinal cancers. This cytokine expression of IL-12 is designed to increase the local 

concentration of IL-12 with minimal escape into the systemic circulation thus minimizing the systemic toxicities associated with 

the recombinant IL-12 therapy. Two Phase I trials of EGEN-001 administered as a single agent or in combination with standard 

chemotherapy agents in advanced ovarian cancer patients have been completed, a Phase II trial of EGEN-001 as a single agent in the 

platinum-resistant patient population is in progress, and a Phase I/II trial in combination with chemotherapy agents in metastatic 

colorectal cancer patients has been initiated. The manufacturing of this investigational product has been successfully advanced 

from bench scale to clinical scale and several cGMP lots have been successfully produced. A detailed description of EGEN-001 

pharmacology/toxicology and pharmaceutical properties will be presented. 



1:15 pm - 2:45 pm 


Topical Review: Navigating a Dificult Funding Climate 

This session will present NIH training and career opportunities for investigators as well as what type of support is available from 

Foundations and Industry. In addition, two presentations will focus on how to write an application from the perspective of a senior and 

junior investigator. 

CO-CHAIRS: Scott Q. Harper, PhD and Sonia I. Skarlatos, PhD 

SPEAKERS 



Startup Funds Don’t Last Forever: A Junior PI’s Path Toward Funding and Growing a New Lab 

Many of us spend a decade or more training to become independent scientists. When the opportunity inally arrives, walking into 

our own empty labs for the very irst time can be exhilarating and overwhelming. This moment also means the clock begins ticking 

on the next stage of our careers. Arguably the most critical part of this next stage is acquiring independent funding to sustain and 

grow our lab, which is no easy task in today’s economic climate. In this presentation, I will discuss my path for leveraging my lab 

startup package into independent funding. 

Drew E. Carlson, PhD 

Fellowships and Career Awards: Stepping Stones to Research Independence 

The NIH offers a myriad of fellowships and K awards to facilitate the pursuit of a career in biomedical research. Understanding the 

objectives, eligibility requirements, review criteria, and process in selecting and applying for these funding opportunities is central 

to the submission of a competitive proposal. This presentation will focus on the general principles that apply to these mechanisms 

and how to navigate the intricacies of the NIH to identify the best options for your career stage and scientiic expertise. 


Funding from Foundations: Perspectives from the Foundation Fighting Blindness 

The Foundation Fighting Blindness (FFB) is the largest non-governmental supporter of research related to inherited retinal 

degenerations. The goal of this presentation is to describe the types of research funding available from FFB, which range from 

Career Development Awards to Individual Investigator Awards to Research Center grants. The grant application process and review 

processes will be described, with an emphasis on practical advice about preparing competitive proposals for foundations in general, 

using FFB as an example. 


41




1:15 pm - 2:45 pm 

ROOM: 150 ABC 

Topical Review: Synthetic Gene Delivery 

An overview of different philosophies of synthetic delivery systems for nucleic acids. The irst talk will address silk elastin-like proteins for 

gene delivery and how they can be combined with virus for an enhanced hybrid gene delivery vehicle. The second talk will focus on “smart” 

pH-sensitive polymeric carriers for therapeutic delivery of nucleic acids. The inal talk will examine cell-penetrating peptides for retinal gene 

delivery. 

CHAIR: James Yockman, PhD 

SPEAKERS 


Recombinant Polymers for Gene Delivery 

Polymeric systems have shown utility in gene delivery. They can form complexes with nucleic acids or be used as matrices for 

controlled release. Majority of polymers used for such purposes are synthesized by chemical strategies that result in limited control 

over polymer structure and therefore function. Recombinant polymers provide exquisite control over backbone structure. This talk 

will summarize the use of recombinant polymers for gene delivery applications. 

You Han Bae, PhD 

pH-Sensitive Polymeric Carrier for Nucleic Acid Delivery 

The journey of a gene carrier from an administration site to target individual cells encounters a series of formidable barriers in 

the blood compartment, when crossing vascular walls, and while penetrating extracellular matrix. Cell uptake and intracellular 

traficking steps then follow, which have been major foci in carrier development and in vitro tests. The carrier may require 

switching surface property depending on environmental conditions during the voyage for higher probability of reaching its target 

cell and action site. The potential roles of pH-responsive polymers, particularly weakly anionic polysulfonamide and cationic 

poly(L-histidine), in this challenging journey will be discussed. 

Rajendra Kumar-Singh, PhD 

Pegylated Peptide-DNA Nanoparticles Delay the Onset of Retinal Degeneration in Mouse Models of Retinitis 

Pigmentosa 

We have developed a novel peptide referred to as ‘Peptide for Ocular Delivery’ or POD that can penetrate cells and tissues in vivo 

within 5 minutes. When pegylated, POD can compact DNA and enable marker or therapeutic gene delivery to retina in vivo. These 

nanoparticles are approximately 150 nm in size and can enable therapeutic transgene expression in vivo for more than 70 days. 

POD-DNA nanoparticles can reduce apoptosis and delay retinal degeneration in a mouse model of retinitis pigmentosa. POD has 

potential therapeutic applications in ocular as well as non ocular organ systems. 


1:15 pm - 2:45 pm 


Topical Review: The Ins and Outs of Integration Deicient Lentiviral Vectors (IDLV’s) 

The Ins and Outs of Integration Deicient Lentiviral Vectors (IDLV’s)”, will focus on the development and utilization of IDLV’s in preclinical 

trials. The session comprises three presentations. The irst is an introductory presentation, which will concentrate on the molecular 

mechanisms involved in the production and regulation of gene expression from IDLV’s. The second presentation will focus on illegitimate 

integration of IDLV’s, and the third presentation will describe the development of a novel IDLV system for cancer immunotherapy. 

CHAIR: Tal Kafri, MD, PhD 

SPEAKERS 

Tal Kafri, MD, PhD 

The Basics of Integration Defective Lentiviral Vectors 

This introductory presentation will focus on the molecular mechanism involved in the generation of integration defective lentiviral 

vectors (IDLV’s) and the molecular assays employed to determine the eficacy and safety of the currently used IDLV gene delivery 

systems. In addition the presentation will outline advancement in IDLV design, which improve IDLV gene expression and reduce 

illegitimate integration. 

42 



Manfred Schmidt, PhD 

Clonality and Biosafety Aspects of Illegitimate Integration 

Integrating retroviral vectors have been used successfully in clinical applications. With the occurrence of integration associated 

severe side effects, enhanced efforts have been set for the clinical translation of integrase deicient viral vectors to guarantee 

improved biosafety. In this presentation, the focus is set on studies performed for assessing frequency, proile and mechanisms of 

(illegitimate) integration. 

Scott H. Robbins, PhD 

Development of an ID-LV as a Gene Delivery Platform for Immunotherapy Applications 

One of the most fundamental challenges for the design of next generation vaccines is the generation of antigen-speciic CD8 T 

cell responses. The direct administration of lentivector-based vaccines genetically encoding antigen is proving to be an effective 

approach to elicit CD8 T cell responses in pre-clinical animal models. The ability to alter the tropism of lentiviral vectors via 

pseudotyping and other mechanisms makes them attractive as a platform technology on which to base product candidates for 

clinical applications such as antigen-directed immunotherapy and vaccines. Examples of the functionality of integration-competent 

and -deicient lentiviral vectors as antigen delivery vehicles in pre-clinical settings will be provided as well as an example of the 

design of a dendritic cell-directed integration-deicient lentiviral vector that is currently under development as an antigen-directed 

cancer immunotherapy (a “cancer vaccine”). 


Oral Abstract Session 130 

3:15 pm - 5:15 pm 

ROOM: BALLROOM A 

AAV Vector Development & Application 

CO-CHAIRS: Mavis Agbandje-McKenna, PhD and Hiroyuki Nakai, MD, PhD 

(Abstracts 1 through 8; see page 87) 


3:15 pm - 5:15 pm 


Cancer - Immunotherapy I 

CO-CHAIRS: Malcolm K. Brenner, MD, PhD and John J. Nemunaitis, MD 

(Abstracts 9 through 16; see pages 87-88) 


3:15 pm - 5:15 pm 


Cancer - Oncolytic Viruses 

CO-CHAIRS: David Kirn, MD and Stephen J. Russell, MD, PhD 



3:15 pm - 5:15 pm 


Advancing the Science of Gene Therapy for Hematologic Diseases 

CO-CHAIRS: Arnab Ghosh, MD, PhD and Donald B. Kohn, MD 



43




3:15 pm - 5:15 pm 


Neurological Disorders: Monogeneic Disorders 

CO-CHAIRS: Raymond T. Bartus, PhD and Nicholas Boulis, MD 



3:15 pm - 4:15 pm 


Chemical and Molecular Conjugates 

CO-CHAIRS: Young Jik Kwon, PhD and Assem Ziady, PhD 



3:15 pm - 4:15 pm 

ROOM: 150 ABC 


CO-CHAIRS: Renata Stripecke, PhD and Grant D. Trobridge, PhD 



4:20 pm - 5:20 pm 


Cell Processing and Vector Production 

CO-CHAIRS: Adrian P. Gee, PhD and Isabelle Riviere, PhD 



4:20 pm - 5:20 pm 

ROOM: 150 ABC 

Sensory (Ophthalmic & Auditory) Gene & Cell Therapy 

CO-CHAIRS: Alberto Auricchio, MD and Tonia S. Rex, PhD 


Exhibit Hall Welcome Reception 

5:15 pm - 7:15 pm 

ROOM: EXHIBIT HALL C/D 

44 



Workshop 140 

7:30 pm - 9:30 pm 

Room: 150 DEFG 

Commercialization Workshop 

CO-CHAIRS: Estuardo Aguilar-Cordova and Brendan Lee, MD, PhD 

SPEAKERS 

Peter Kolchinsky 

What makes Investors want to Invest 

Nick Leschly 

Who Utilizes Early Phase Resources 


Fabrizia Bignami, PhD 

What is Big Pharma Looking for in Gene or Cell Therapy 

Suzannah Sundby, JD 

What IP Issues are Relevant for Successfully Commercializing Gene and Cell Therapies 

Gene and cell therapies are different from classic small molecules in that intellectual property issues can evolve and become 

increasingly complex as scaling up from the laboratory setting and commercialization proceeds. What effect do modiications 

and optimizations to vector designs and manufacturing processes, including cell isolation and culturing techniques, have on one’s 

right to make, use, and sell the intellectual property Do you still own the rights to the intellectual property Will the intellectual 

property rights of another prevent you from commercializing your own product Ms. Sundby will discuss IP in the cell and gene 

therapy space with particular attention paid to what questions an investigator or institution should consider prior to investing 

further resources in pipeline development. 

New Member Welcome Reception 

7:30 pm - 8:30 pm 

ROOM: 255 B 


45

Program Schedule, Thursday, May 16, 2013 

George Stamatoyannopoulos Lecture 200 

8:30 am - 9:35 am 

ROOM: BALLROOM E-J 

George Stamatoyannopoulos Lecture 

CHAIR 

SPEAKER 



Mario R. Capecchi, PhD 

Modeling of Human Disease in the Mouse in the 21st Century: From Cancer to Neuropsychiatric Disorders 

Gene targeting allows the designed modiication of any gene in the mouse genome. Since genes impact all biological phenomena 

this methodology can be used to study any biological phenomena common to mammals in the mouse. We are using it to model 

human disease in the mouse. The models can be used to analyze the pathology of the disease at a level not feasible in humans and 

as a platform for the development of new therapeutic protocols. I will discuss modeling of a neuropsychiatric disorder, obsessive 

compulsive (OCD) spectrum disorder in the mouse. These studies provide the unexpected conclusion that microglia, the immune 

cells of the brain derived from bone marrow, are controlling behavior in the mouse. 

Exhibit Hall Coffee Social 

9:35 am - 10:30 am 



10:30 am - 12:30 pm 


50 Years of Research in Alpha-1 Antitrypsin 

CHAIR: Terence R. Flotte, MD 

SPEAKERS 

Robert A. Sandhaus, MD, PhD 

Historical Review of the Past 50 Years of Research on Alpha-1 Antitrypsin Disorder 

First described 50 years ago, Alpha-1 Antitrypsin Deiciency (AATD) was soon associated with the development of both lung and 

liver diseases. The rapidity with which the biochemical, pathophysiologic, epidemiologic, and therapeutic advances followed this 

initial discovery has been astounding, even by today’s standards. In spite of this, there is still much to be learned about the alpha-1 

antitrypsin protein and its roles in health and disease. 


Gene Therapy for Alpha 1-antitrypsin Deiciency with AAV Intrapleural Administration 

a1-antitrypsin (a1AT) deiciency, caused by SERPINA1 gene mutations, results in low a1AT serum levels and development of earlyonset 

panacinar emphysema, reducing life span by 15 to 20 years. Current treatment is weekly intravenous administration of a1AT 

puriied from pooled human plasma. Adeno-associated (AAV)-mediated delivery of a1AT is an ideal strategy for gene therapy for 

the lung manifestations of a1AT deiciency because AAV vectors mediate sustained transgene expression. We have established a 

novel, highly eficient strategy for gene therapy for a1AT deiciency based on intrapleural administration of AAV serotype rh.10 

coding for human a1AT. The advantage this approach offers is proximity of vector expression to the site of action. Pre-clinical 

studies comparing intrapleural administration of 25 AAV serotype vectors demonstrated that the highest a1AT levels were achieved 

with AAVrh.10, a serotype that is derived from a non-human primate, minimizing the challenge of pre-existing human anti-vector 

immunity limiting persistent gene expression. Safety studies in mice and non-human primates have shown this strategy is safe 

and will provide sustained expression a1AT. Our overall hypothesis is that a single intrapleural administration of an AAVrh.10 

gene transfer vector coding for the human a1AT cDNA will provide sustained therapeutic levels in the lung suficient to treat a1AT 

deiciency. 

46 



Christian Mueller, PhD 

MicroRNA’s and Alpha-1 Antitrypsin Deiciency 

Terence R. Flotte, MD 

Advances in Clinical Gene Therapy Trials for Alpha-1 Anti-trypsin Deiciency 


10:30 am - 12:30 pm 


Clinical Updates of Stem Cell Therapies 

CO-CHAIRS: Jane S. Lebkowski, PhD and Eirini P. Papapetrou, MD, PhD 

SPEAKERS 

Michele De Luca, MD 

Corneal Regeneration By Cultured Limbal Stem Cells 

Adult stem cells are cells with a high capacity for self-renewal that can produce terminally differentiated progeny. Stem cells 

generate an intermediate population of committed progenitors, often referred to as transit amplifying (TA) cells, that terminally 

differentiate after a limited number of cell divisions. Human keratinocyte stem cells are clonogenic and are known as holoclones. 

Human corneal stem cells are segregated in the limbus while limbal-derived TA cells form the corneal epithelium. Cultivated limbal 

stem cells generate sheets of corneal epithelium suitable for clinical application. We report long-term (up to 15 years) clinical 

results obtained in an homogeneous group of patients presenting with corneal opaciication and visual loss due to chemical burndependent 

limbal stem cell deiciency. The corneal epithelium and the visual acuity of these patients have been restored by grafts of 

autologous cultured limbal keratinocytes. In post hoc analyses, success was associated with the percentage of p63-bright holocloneforming 

stem cells in culture, but not with total number of clonogenic cells, colony size or growth rate of the cultures. Graft failure 

was also associated with the type of initial ocular damage and postoperative complications. 

Joshua Hare, MD 

Cell Therapy for Chronic Ischemic Heart Disease from Concept to Clinic 


Brian W. Bigger, PhD 

Hematopoietic Stem Cell Gene Therapy for Neurological Diseases 

Haematopoietic stem cell transplantation (HSCT) has traditionally been used to treat a select group of single gene neurological 

diseases, with particular success in the neurodegenerative lysosomal disease Hurler syndrome. In many lysosomal diseases 

exogenous enzyme can be taken up by, and cross-correct affected cells, but the blood brain barrier excludes enzyme from the 

brain. Neurological correction after HSCT relies on monocyte/microglial donor cell traficking into the brain to deliver enzyme, and 

enzyme dose is thought to be one of the factors limiting HSCT to a relatively narrow range of neurological indications. Autologous 

haematopoietic stem cell gene therapy, using retroviral or lentiviral vectors to overexpress enzyme in ex vivo transduced and 

transplanted cells has been used to great effect in X-SCID and other immunodeiciencies and more recently has been adopted as a 

potential treatment for neurological lysosomal and related diseases. In this talk the initial clinical trial results in this ield will be 

discussed, together with the current challenges faced by this therapeutic approach, what is being done to address them and where 

the future for this exciting therapy lies. 

Gary K. Steinberg, MD, PhD 

Stem Cell Therapy for Stroke 

No treatment currently exists to restore lost brain function after stroke. Animal studies have demonstrated that post-ischemic 

intraparenchymal, intravenous and intra-arterial administration of various types of stem cells can improve neurologic outcome. 

Although some integration of neural stem cells into the peri-infarct brain has been shown, the main mechanisms underlying 

recovery of function are likely secretion of trophic factors and immunomodulation that enhance endogenous plasticity and 

recovery. These native repair processes include axonal sprouting, dendritic branching, neovascularization, neurogenesis, 

gliogenesis, synaptogenesis and decreased inlammation. To date 11 trials of cellular therapy for stroke have been completed 

and more than 15 additional trials are underway. This talk will briely review the experimental basis leading to the clinical trials, 

and focus on summarizing the clinical results of published and ongoing stroke trials using neural, bone marrow derived and 

other stem cells. Unresolved issues will be discussed, including the best cell source and type, optimal route of delivery, timing of 

transplantation and patient selection. 


47



10:30 am - 12:30 pm 


Frontiers of Cardiovascular Gene and Cell Therapy 

CO-CHAIRS: David A. Dichek, MD and Michael J. Passineau, PhD 

SPEAKERS 

Charles R. Bridges, MD, ScD 

Challenges of Gene and Cell Therapy to the Heart 

In this presentation I will describe the main challenges of current cardiac cell and gene therapy with an emphasis on challenges to 

eficient delivery. I will give a detailed comparison of available gene and cell delivery techniques. I will discuss documented and 

potential complications of cell injections and gene transfer such as the risk of arrhythmias, incidence of coronary restenosis and 

malignancy. Finally, I will focus on the most recent results of cardiac cell and gene therapy trials and the major lessons drawn from 

these trials. 



Vascular MiRNA and Pulmonary Hypertension 

MicroRNAs (miRNAs) are short non-coding RNAs which post-transcriptionally regulate gene expression. miRNAs are important 

in governing cell differentiation, development and disease. We have investigated the role or individual miRNAs in pathological 

vascular remodeling, as well as the potential therapeutic value of manipulating selected miRNA in vivo. I will discuss these 

indings with emphasis on vascular remodeling associated with both venous and arterial remodeling, i.e. vein graft failure and the 

development of pulmonary arterial hypertension. 

Marc Penn, MD, PhD 

Gene Therapy for Heart Failure 

The success of establishing antegrade low in patients with acute myocardial infarction has led to signiicant advancements in the 

survival of patients with acute ischemic events of the myocardium. Unfortunately this success had led to a signiicant increase in 

the prevalence of heart failure. To address this need several investigators have turned to gene therapy as a strategy to either repair, 

restore or optimize cardiac function. Excitingly these strategies have entered clinical trials with encouraging results. We will discuss 

the strategies currently under investigation as well as those that maybe entering the clinical realm in the future. 

Doris A. Taylor, PhD 

Personalizing Cell Therapy: Initial CCTN Experience 


10:30 am - 12:30 pm 


New Paradigms for Immune Cell Therapies 

CO-CHAIRS: Cynthia Dunbar, MD and Paris Margaritis, DPhil 

SPEAKERS 

David L. Porter, MD 

Chimeric Antigen Receptor Therapies for Lymphoid Malignancies 

This presentation will describe the biological activity and outcomes of patients treated with CART19 (CTL019) cells; these are 

autologous T cells collected by leukapheresis and transduced with a lentivirus encoding anti-CD19 scFv linked to 4-1BB and CD3-z 

signaling domains. Gene-modiied T cells can then target CD19+ cells. Cells are expanded and activated ex-vivo by exposure to anti- 

CD3/CD28 beads prior to infusion. Patients are treated for advanced and/or refractory CLL or ALL. We previously reported marked 

in-vivo expansion, long-term persistence and effective anti-tumor activity of CART19 cells in 3 patients with CLL with relatively 

short follow up (Porter, et al NEJM 2011; Kalos et al Sci Trans Med 2011). Results from the clinical trials will be updated with longterm 

follow-up and include presentation of data from a larger cohort of patients. We show that these cells can undergo massive 

in-vivo expansion, persist for more than 2 years and remain biologically active, and can induce potent and sustained responses for 

patients with advanced, refractory and high risk CLL and relapsed, refractory ALL. 

Maria-Grazia Roncarolo, MD, PhD 

Cell Therapy with Regulatory T Cells to Dampen Undesired Immune Responses 

48 



Nicholas Restifo, MD 

Curative Cancer Immunotherapy Using Stem cell-like T cells 

T lymphocytes play a critical role in the immune response against infectious disease and cancer. The recently identiied T memory 

stem cells (TSCM) are endowed with the stem cell–like attributes of self-renewal capacity and multipotency. These qualities make 

TSCM an attractive subset to employ in adoptive immunotherapies for cancer because they might overcome current limitations, 

including ineficient T-cell engraftment, poor persistence, and inability to mediate a prolonged immune attack. TSCM display robust 

proliferative and survival capacities and were superior to other memory T-cell subsets in causing regression of large, established 

tumors in preclinical models. Protocols to facilitate translation of TSCM into the clinic will be discussed. 

Jeffrey S. Miller, MD 

Educating Natural Killer Cells to Kill Cancer 

Natural killer (NK) cells express a number of activating and inhibitory receptors to determine their function through a process 

called NK cell education. We have shown that adoptive transfer of haploidentical NK cells can induce remissions in some 

patients with refractory acute myeloid leukemia (AML). Limitations of this therapy include host immune rejection, how to 

maximize the persistence and expansion of donor NK cells and overcoming regulatory T-cell suppression. Lastly, donor Killer-cell 

Immunoglobulin-like Receptor (KIR) genes may inluence NK cell activity to enhance anti-tumor eficacy. Understanding the role of 

KIR and other receptors may allow us to exploit adoptive transfer of NK cells for therapeutic use in patients with advanced cancers. 


10:30 am - 12:30 pm 


Recent Advancements in the Non-Viral Delivery of Nucleic Acid Based Drugs 

CO-CHAIRS: Ian MacLachlan, PhD and Olivia Merkel, PhD 

SPEAKERS 


Combinatorial Development of Synthetic Nucleic Acid Delivery Systems 


Jianjun Cheng, PhD 

a-Helical Polypeptides for Non-Viral Gene Delivery 

We designed a class of helical, cationic charged polypeptides via the ring-opening polymerization of vinyl benzyl glutamic acid 

N-carboxyanhydrides. These materials have unusual helical stability and display excellent cell penetrating property outperforming 

TAT peptide. By controlling the chemical, morphological and self-assembly structures of these side chain charged, helical 

polypeptides, we identiied a few materials that showed excellent gene/siRNA delivery eficiency in vitro and in vivo. 

Thomas Knotts, PhD 

Protein Structure and How it Changes in Association with Surfaces 

Proteins/surface interactions are important in a variety of existing and emerging biomedical techniques and devices. It has been 

known for decades that surfaces can drastically change the structure of attached or adsorbed proteins, but a priori prediction of the 

changes is not currently possible. In an attempt to develop some predictive capabilities with respect to protein/surface interactions, 

we have used advanced molecular simulation to test several theories as to how protein folding mechanisms and stabilities are 

affected by surfaces. This talk will present many of these results, and more importantly, design heuristics that can be used to ensure 

proteins of interest are stable, and functional on surfaces. Taken as a whole, the results offer compelling evidence that the rational 

design of technologies involving proteins interacting with surfaces is possible. 


Silk-elastinlike Polymers for Matrix-mediated Gene Delivery 


49



10:30 am - 12:30 pm 


The GTRP: Perspectives of an Investigator and the Program Cores 

CO-CHAIRS: Cheryl L. McDonald, MD and Sonia I. Skarlatos, PhD 

SPEAKERS 


Daniel Rader, MD 

How the GTRP Facilitates Translational Research: An Investigator’s Perspective 

Atherosclerosis is a complex and heritable disease intimately inluenced by lipoprotein metabolism. Unbiased genome-wide 

association studies (GWAS) have identiied dozens of novel loci robustly associated with both plasma lipid traits and atherosclerotic 

coronary artery disease (CAD). Several of the genes at these loci could represent potential novel therapeutic targets for the 

treatment of inherited dyslipidemias and the prevention or treatment of CAD. Adeno-associated viral (AAV) vectors are a valuable 

tool for effective and stable in vivo expression of cDNAs in relevant animal models of disease. Through support from the GTRP, we 

have used AAV8 vectors to express a number of potential therapeutic genes at lipid/CAD GWAS loci in the livers of ‘humanized’ 

mice. This systematic approach has not only yielded important new biological information about the effects of novel GWAS genes on 

lipid metabolism and atherosclerosis, but also has placed a number of new ‘therapeutic’ genes on the map. This classic translational 

approach moving from unbiased human genetics to mechanisms and therapeutic effects in model systems is made possible by the 

support of the GTRP. 


GTRP: Facilitating Bench to Bedside 

The purpose of the GTRP is to provide researchers with resources that are critical to advancing investigational gene therapy 

products into clinical testing. Services along the product development spectrum – ranging from production of preclinical vectors to 

assistance with early-phase clinical trials – are offered through the Program at no cost to the investigator. The Program primarily 

supports research in heart, lung, and blood diseases. 


A Toolkit of Vectors for Gene Therapy 

The Preclinical Vector Core provides high quality viral vectors based on adeno-associated virus (AAV), adenovirus and lentivirus 

as well as non-viral vectors for basic research directed toward clinical application. Also offered are a host of other services such as 

consultation on vector construction and experimental design, quality control services appropriate for vectors destined for use in 

small or large animal models, immunology testing and preclinical biodistribution analysis. Of particular importance to NHLBI GTRP 

investigators has been distribution through the program of novel serotype AAV vectors which have become the gold standards in 

the ield for mediating gene delivery to heart, lung and liver. In addition to offering a variety of vector platforms, the Preclinical 

Core rapidly transitions new technologies from key laboratories for distribution as well as develops reagents and maintains a large 

inventory of vectors in order to provide GTRP participants with a veritable vector toolbox for advancing preclinical studies toward 

clinical translation. 

Janet Benson, PhD, DABT 

From Mice to Man: IND-enabling Preclinical Studies 

The mission of the Pharmacology/Toxicology (Pharm/Tox) Core is to aid investigators in the design and implementation of INDenabling 

preclinical safety and biodistribution studies. This is often one of the most challenging and costly steps in the translational 

process. The Pharm/Tox Core scientists work closely with the Investigator(s), the GTRP Core providing the vector (if applicable), 

and the GTRP Clinical Coordinating Center to facilitate development of the IND packages and the pre-clinical study protocol. The 

GTRP Pharm/Tox Core provides dosing, endpoint evaluations including but not limited to: clinical observations, clinical pathology, 

histopathology, safety pharmacology, vector biodistribution (qPCR), immunology endpoints, as well as assessments of transgene 

expression. Upon completion of the study, the Pharm/Tox Core staff prepares an audited inal report for inclusion in the IND 

submission to the FDA. 

50 



J. Fraser Wright, PhD 

AAV Vectors for Clinical Trials: Challenges, and Strategies for Success 

Recombinant viral vectors based on adeno-associated viruses (AAV) have shown tremendous promise for clinical therapeutic 

gene delivery, and an acceleration of gene-based product development programs has been catalyzed by recent clinical successes. 

This presentation will describe strategies for advancing AAV-based investigational product chemistry, manufacturing and 

controls to effectively support the translational pathway from IND-supporting pre-clinical studies through clinical studies. An 

important requirement for successful translational research includes well deined manufacturing processes capable of reliable and 

reproducible yields vector of high purity, potency and safety. Another critical requirement is the set of analytical methods required 

to ensure comprehensive investigational product characterization and quality control testing. In this presentation the vector 

manufacturing processes and characterization methods used to support AAV vector-based translational research supported by 

GTRP will be presented. 


Lentiviral Vectors: Can We Move Past Phase I/II 

Lentiviral vectors have advanced into early Phase clinical trials in the setting of genetic diseases, cancer, and other applications 

where integration and long-term expression is required. Compared to traditional integrating vectors, the methods used in 

manufacturing lentiviral vectors are well suited to rapid implementation into Phase I clinical trials. Unfortunately, this property can 

present challenges to generating a licensed product. This talk will discuss the current status of lentiviral vectors and highlight the 

challenges of moving these vectors from research tools to an FDA approved agent. 

Susan B. Sepelak, MS, MSM 

Regulatory Requirements: Navigation is Easier with Assistance 

The GTRP Clinical Coordinating Center provides assistance to investigators in navigating the regulatory processes in translational 

gene therapy research. For approved requests, assistance may be provided directly to the Investigator - assistance ranging from 

help with scheduling pre-preIND meetings with the FDA through organization, compilation and submission of IND applications. 

Regulatory support with clinical site-related issues may also be requested by Investigators to help site staff and Study Coordinators 

prepare for study implementation and manage ongoing regulatory requirements. Investigators who receive clinical trial funding 

assistance through the GTRP may, additionally, apply for support with site selection, study monitoring, and preparation of NHLBIrequired 

study reports. 



12:30 pm - 2:00 pm 

Meet-the-Investigator Lunch Session 220 

12:30 pm - 2:00 pm 

ROOM: 251 E 

New Frontiers – Hemoglobinopathies 

SPEAKERS 




12:30 pm - 2:00 pm 

MEET AT THE SOUTH ENTRANCE OF THE SALT PALACE CONVENTION CENTER 

Huntsman Cancer Institute Tour 

Boxed lunches will be available at the South Entrance shortly before departure to the Huntsman Cancer Institute. 


51



2:00 pm - 4:00 pm 


Clinical Gene & Cell Therapy 

CO-CHAIRS: Carl H. June, MD and Harry L. Malech, MD 



2:00 pm - 4:00 pm 



CO-CHAIRS: Brian D. Brown, PhD and Jennifer E. Adair, PhD 




2:00 pm - 4:00 pm 


AAV Vector Biology 

CO-CHAIRS: Casey A. Maguire, PhD and David V. Schaffer, PhD 



2:00 pm - 4:00 pm 


Stem Cell Engineering and Therapy 

CO-CHAIRS: Hans-Peter Kiem, MD, FACP and Eirini P. Papapetrou, MD, PhD 

(Abstracts 80 through 86; Abstract 79 Withdrawn and Abstract 470 moved from Poster Session II; see pages 93-94) 


2:00 pm - 4:00 pm 


Musculo-skeletal Gene & Cell Therapy I 

CO-CHAIRS: Charles A. Gersbach, PhD and Louise R. Rodino-Klapac, PhD 



2:00 pm - 3:00 pm 

ROOM: 150 ABC 

Cardiovascular Gene & Cell Therapy 

CO-CHAIRS: Dongsheng Duan, PhD and Joseph E. Rabinowitz, PhD 


52 




2:00 pm - 3:15 pm 



CO-CHAIRS: King F. Kwong, MD and Kevin G. Rice, PhD 



3:05 pm - 4:05 pm 

ROOM: 150 ABC 

Evolution of Pulmonary Treatments 

CO-CHAIRS: Uta Griesenbach, PhD and Katherine J. Excoffon, PhD 

(Abstracts 104 through 107; Abstract 105 Withdrawn and Abstract 432 moved from Poster Session II; see pages 95-96) 


3:20 pm - 4:05 pm 


Immunologic & Host Responses in Gene & Cell Therapy 

CO-CHAIRS: Antonia Follenzi, MD, PhD and Federico Mingozzi, PhD 



Exhibit Hall Open: Networking Reception & Poster Session I 

4:00 pm - 6:00 pm 


AAV Vectors - I 

(Abstracts 111-125; see pages 96-97) 

Adenovirus and Other DNA Virus Vectors I 

(Abstracts 126-138; and Abstract 561 moved from Poster Session III; see pages 97-98) 




(Abstracts 155-165; Abstract 165 has been withdrawn from presentation; see page 99) 





Cancer - Immunotherapy I 

(Abstracts 197-209; Abstract 196 withdrawn; see pages 101-102) 

Cancer-Targeted Gene & Cell Therapy: Viral Vector / siRNA-Mediated Therapies 



53


Sensory (Ophthalmic and Auditory) Gene & Cell Therapy 

(Abstracts 222-232; see page 103) 



Fetal and Adult Stem Cells 

(Abstracts 247-258; Abstract 252 has been withdrawn from presentation; see pages 104-105) 



Late Breaking Abstracts I 

(Abstracts 677-700; Abstracts 687 and 698 Withdrawn from Presentation; see pages 105-107) 


Canadian Attendee Reception 

6:00 pm - 7:00 pm 

ROOM: 255 B 

Foundation Symposium 240 

6:00 pm - 8:00 pm 


Gene and Cell Therapy for Cancer 

Supported by the Alliance for Cancer Gene Therapy. 

CO-CHAIRS: Xandra O. Breakeield, PhD and Savio L.C. Woo, PhD 

SPEAKERS 

Joseph C. Glorioso, III, PhD 

Targeted Cancer Therapies 

Cancer cell genetic analyses have identiied driver mutations that dysregulate cell proliferation- and differentiation-related 

pathways, providing focal points for targeting of cancer therapy. At least six targeting strategies have been employed: (i) the use of 

drugs that block different steps in cancer causing signaling cascades leading to the arrest of tumor cell growth and the induction of 

senescence, apoptosis and autophagy, (ii) application of drugs or immune mediators that attack the tumor microenvironment (iii) 

the use of antibodies that inhibit tumor cell growth by blocking growth factor receptors, (iv) coupling of therapeutics to antibodies, 

nanoparticles or other ligands that recognize tumor-associate antigens (v) immune based strategies to induce anti-tumor immunity, 

reduce immune down regulation or exploit engineered immune acting cells to destroy tumors, and (vi) the use of designer biologics 

such as viruses, bacteria or stem cells to selectively destroy tumors. Some notable successes will be highlighted for each of these 

approaches, however tumor heterogeneity makes it unlikely that any one strategy will succeed. Cancer virotherapy remains a 

very versatile strategy that takes advantage of advances in enhanced vector design to provide the most potent, highly selective 

tumor killers. Current methods to control vector transduction and transcription, evasion of innate immunity and enhanced vector 

replication and spread will be presented. 


Oncolytic Virotherapy 

Oncolytic viruses (OV) selectively destroy neoplastic tissue and it is theoretically possible to engineer or adapt any naturally 

occurring virus for this purpose; key engineering strategies will be reviewed. A spreading intratumoral OV infection can damage 

both infected and uninfected tumor cells; the mechanisms underlying both of these activities will be discussed. Oncolytic viruses 

can be combined with drugs or armed with transgenes to accelerate their spread, modulate their immunogenicity/potency or 

facilitate the noninvasive monitoring of their in vivo fate; illustrative examples will be discussed. Finally, several oncolytic viruses 

are currently being tested in clinical trials and the current status of this work will be summarized. 

54 



Carl H. June, MD 

T Cell Mediated Gene Transfer for Cancer Therapy 

The adoptive transfer of anti-tumor T cells can mediate regression of established metastatic cancers in patients. We have begun a 

series of clinical trials using the transduction of genes encoding chimeric antigen receptors (CARs) or anti-tumor T cell receptors 

(TCR) into normal peripheral lymphocytes for use in adoptive cell transfer. Anti-tumor TCRs have been identiied that recognize 

cancer testis antigens. Autologous cells transduced with genes encoding the anti-NY-ESO-1 TCR cancer-testes antigen mediated 

objective clinical responses in patients with myeloma. An afinity enhanced MAGE A3 TCR caused severe adverse events with 

fatal off-target cardiac toxicity, pointing to serious concerns with the ability of pre-clinical testing to identify off target effects of 

TCRs. Patients with acute pre-B cell leukemia or chronic lymphocytic leukemia exhibited a high objective regression rate using 

T cells transduced with a CD19 CAR. The cellular and molecular mechanisms of T cell destruction of cancers are under active 

investigation. Ongoing studies are evaluating chimeric antibody receptors targeting mesothelin, and EGFRvIII. Together these 

studies demonstrate the power and potential of adoptive T cell immunotherapy to mediate the regression of established advanced 

cancer in humans. 

Lieping Chen, MD, PhD 

New Perspectives in the Development of Cancer Vaccines 

There is ample evidence that human malignancies express tumor-associated or tumor-speciic antigens. Immunization with cancer 

antigen-based vaccine in animal models or patients with advanced cancer induce tumor-reactive T cells in lymphoid organs and 

these T cells can be detected in blood. However, increased circulating tumor-reactive T cells often do not correlate with regression 

of advanced cancer. Recent studies reveal that speciic resistant mechanisms are developed in the tumor microenvironment to 

suppress activity of tumor-iniltrating T cells. It is thus important to understand and identify molecules underlying these resistance 

mechanisms. I will focus my discussion on analysis of mechanisms in the tumor microenvironment in the context of improvement 

of the eficacy of cancer vaccines. 



55



6:00 pm - 8:00 pm 


Association Francaise Contre les Myopathies/Muscular Dystrophy Association Symposium 

Jointly planned by the Association Francaise contre les Myopathies and the Muscular Dystrophy Association. 

CO-CHAIRS: Charles A. Gersbach, PhD and Kathryn R. Wagner, MD, PhD 

SPEAKERS 



Lentiviral Modiication of Myogenic Stem Cells 

Over the past decade adeno-associated viral (AAV) vectors have merged as a potential therapeutic for muscle disorders. While 

AAV vectors can eficiently transduce post-mitotic myoibers, they display low tropism for myogenic stem cells and are rapidly lost 

during cell proliferation due to minimal genomic integration. In contrast, lentiviral vectors can stably transduce myogenic stem cells 

both in vitro and in vivo, enabling expansion and transplantation of genetically modiied cells for therapeutic interventions. This 

lecture will review approaches for modifying myogenic stem cells for in vivo and ex vivo gene therapy of myogenic disorders such as 

the muscular dystrophies. 


Regional Limb Vein Perfusion in Muscular Dystrophy Dogs and Human Patients 

Fulvio Mavilio, PhD 

Gene-based Therapies for Neuromuscular Disorders 

Most inherited neuromuscular diseases have no therapy, and collectively represent a signiicant unmet medical need. Genethon and 

its collaborating clinical centers have launched an ambitious program aimed at developing gene therapy approaches for at least 

three of these diseases (Duchenne muscular dystrophy, myotubular myopathy and spinal muscular atrophy) based on systemic 

or loco-regional delivery of AAV vectors. We will provide an overview of the program and of the scientiic, regulatory and clinical 

challenges associated with its development. 

Anna Buj Bello, MD, PhD 

Myotubularin Deicie ncy 

Myotubular myopathy (XLMTM) is a very severe congenital muscle disorder due to mutations in the MTM1 gene. An overview 

about the disease, animal models and current experimental therapies, in particular our results on intravenous AAV-mediated gene 

replacement therapy, will be presented. Outstanding Achievement Award Lecture 300 

56 



Plenary Session 300 

8:00 am - 9:00 am 


Outstanding Achievement Award Lecture and Presentation of the Distinguished Service Award 

and Special Recognition Award 

CHAIR 


DISTINGUISHED SERVICE AWARD RECIPIENTS 

SPECIAL RECOGNITION AWARD RECIPIENT 

Sonia I. Skarlatos, PhD Catherine McKeon, PhD John J. Rossi, PhD 

SPEAKER 



Deciphering Vector and Non-coding RNA Biology: A New Frontier in Nucleic Acid based Therapies 

In the last decade, we have witnessed great improvements in vector biology and utilized these for novel biomedical discoveries and 

therapeutic applications. In addition, it is now established that over 90% of the genome is transcribed, yet the role for most of the 

non-coding RNAs remains unclear. I will discuss our studies aimed at both uncovering the generation and function of non-coding 

RNAs, as well as the mechanisms important for the development of new vector-based gene transfer approaches to manipulate gene 

expression. Together, these approaches have provided continued and powerful insight into gene therapy mechanisms used to treat 

an array of diseases. 

Exhibit Hall Coffee Social 

9:00 am - 9:45 am 



57

Program Schedule, Friday, May 17, 2013 


9:45 am - 11:45 am 


Cancer Gene and Cell Therapy: Results from Clinical Trials 

CO-CHAIRS: Evanthia Galanis, MD, DSc and Juan F. Vera, MD 

SPEAKERS 

Svend O. Freytag, PhD 

Randomized, Controlled, Phase 2 Trial of Oncolytic Adenovirus-mediated Suicide Gene Therapy Plus IMRT 

Versus IMRT Alone in neWly-diagnosed, Intermediate-risk Prostate Cancer 

We have developed a multi-modal, gene therapy-based approach for the treatment of cancer involving an oncolytic adenovirus, 

suicide gene therapy and radiation therapy. We have evaluated the safety and eficacy of our approach in six clinical trials, including 

a randomized, controlled phase 2 trial in prostate cancer. The results of these clinical studies will be presented and the lessons 

learned discussed. 


Oncolytic Measles Virus Strains in Cancer Treatment Trials: An Update 

Measles virus is a negative strand RNA virus (paramyxovirus) with oncolytic properties. The signiicant preclinical activity of MV- 

Edmonston vaccine strains against multiple different animal tumor models, their potent bystander effect, their selectivity against 

tumor cells, and their ability to retain their oncolytic properties when engineered and retargeted, makes them a promising oncolytic 

platform. Two trackable engineered measles virus strain derivatives are currently in clinical testing; a derivative expressing the 

human carcinoembryonic antigen (MV-CEA) and a strain expressing the human sodium iodine symporter (MV-NIS). Phase I/II trials 

are ongoing or have been completed in ovarian cancer, glioblastoma multiforme, multiple myeloma, mesothelioma and head and 

neck cancer and a phase I/II trial of mesenchymal stem cell delivery will be activated later this year. Clinical data to date, challenges 

in clinical applications of oncolytic measles virus strains and preclinical data supporting future translational directions will be 

discussed. 



CAR Modiied T-cells, From the Bench to the Bedside 

T cells may be genetically modiied to express artiicial T cell receptors, termed chimeric antigen receptors or CARs targeted to 

tumor associated antigens. Previous work in preclinical models has demonstrated the potential of this approach in targeting T 

cells to systemic tumor and in turn eradicating tumor. More recently, we and others have successfully moved this technology to the 

clinical setting demonstrating early very promising responses with CD19 targeted CAR modiied T cells in patients with relapsed 

B cell malignancies including acute lymphoblastic and chronic lymphocytic leukemias as well as indolent B cell non-Hodgkins 

lymphomas. However, limitations to this adoptive T cell approach need to be addressed before this approach may be successfully 

optimized to B cell malignancies and further expanded to other cancers targeting other tumor associated antigens. One such an 

approach is the design of “armored CAR” T cells additionally modiied to express cytokines or co-stimulatory ligands to enhance in 

vivo cytotoxicity as well as persistence of CAR modiied T cells following infusion. 

Stephen Gottschalk, MD 

Cell Therapies for Solid Tumors: Successes and Challenges 

The outcome for patients with advanced solid tumors has not signiicantly improved over the last 2 decades. Thus, new targeted 

therapies are needed to improve current outcomes, and T-cell immunotherapy has the potential to fulill this need. In my talk I will 

review i) the use of Epstein-Barr virus (EBV)-speciic T cells to treat patients with advanced stage EBV+ nasopharyngeal carcinoma, 

ii) our recent studies to target solid tumors and their supporting stroma with chimeric antigen receptor (CAR) T cells, and iii) novel 

approaches to render T cells speciic for solid tumor antigens. 

58 




9:45 am - 11:45 am 


Cell and Tissue Engineering for Musculoskeletal Disease 

CO-CHAIRS: Alan J. Nixon, DVM, MS and Bing Wang, MD, PhD 

SPEAKERS 

Rita Perlingeiro, PhD 

From iPS cells to Skeletal Muscle Precursors: Potential for Gene Correction and Therapeutic Application 

A major obstacle in the application of cell-based therapies for the treatment of neuromuscular disorders is obtaining the 

appropriate number of stem/progenitor cells to produce effective engraftment. The use of pluripotent embryonic stem (ES) or 

induced pluripotent stem (iPS) cells could overcome this hurdle. We have developed a method to eficiently generate skeletal 

myogenic progenitors from pluripotent stem cells through Pax3 or Pax7 induction. We have applied this method to mouse ES 

and iPS cells, and more recently to human ES and iPS cells. In each case, Pax7-induced cells engraft extensively and promote 

improved contractility in transplanted dystrophic muscles (using a mouse model for Duchenne Muscular Dystrophy). In addition 

to generating new myoibers, the transplanted cells also seeded the muscle satellite cell compartment. Accordingly, in long-term 

cohorts, the frequency of human-speciic dystrophin+ ibers declined only modestly at one year post-transplant, compared to one 

month. Based on these indings, we are now focusing on the genetic correction of iPS cells encoding mutations in the dystrophin 

gene. Such studies are crucial for the future therapeutic application of iPS-derived myogenic progenitors in the autologous setting 

for DMD. These more recent studies will also be discussed. 

Rocky S. Tuan, PhD 

Regenerative Applications of Adult Stem Cells 

Farshid Guilak, PhD 

Engineering Functional Tissue Replacements to Treat Osteoarthritis 

Osteoarthritis is painful and debilitating joint disease that is characterized by pain and degenerative changes in the articular 

cartilage and other joint tissues. While a number of techniques have been developed over the years for the treatment of small 

cartilage defects, there have been few attempts at tissue-engineered therapies for end-stage osteoarthritis. Some of the major 

considerations for this approach include the identiication and characterization of an abundant and accessible cell source as well as 

the design of biologically and mechanically functional scaffolds that can withstand joint loading. Here we describe the engineering 

of large, anatomically-shaped cartilage constructs using different stem cell sources (adipose stem cells, mesenchymal stem cells, 

or induced pluripotent stem cells), combined with mechanically functional cell-instructive scaffolds based on three-dimensional 

weaving of biocompatible ibers. As each iber is selected individually, this textile processing technique allows site-speciic 

functionalization of proteins or virus to facilitate the formation of complex inhomogenous tissues from a single cell source. 

Brian Johnstone, PhD 

Stem Cells for Cartilage Repair 


9:45 am - 11:45 am 


Gene Therapy and Regenerative Medicine for Ocular and Metabolic Diseases 

CO-CHAIRS: Alberto Auricchio, MD and Thierry C. VandenDriessche, PhD 

SPEAKERS 


Ian Constable, AO 

Development of AAV-sFLT-1 for the Treatment of Wet Age-related Macular Degeneration 

The eye is an attractive organ in which to develop gene therapy. There is relative ease of access, a benign immunological response 

and the availability of high resolution non-invasive structural and functional tests. The safety and possible eficacy of Adenoassociated 

Viral (AAV) gene therapy is now well established in young humans with RPE-65 defective retinal blindness. We have 

adopted similar techniques to treat wet macular degeneration by subretinal injection of AAV-sFLT-1 to up-regulate production of 

sFLT-1, a competitive analogue of the soluble Vascular Endothelial Growth Factor receptor. 


59


Ludovic Vallier, PhD 

Gene Correction of AAT Deiciency Using iPS-derived Hepatic Cells 

The possibility of generating human Induced Pluripotent Stem Cells from somatic cells using epigenetic reprogramming represents 

a unique opportunity for personalised regenerative medicine. Indeed, these pluripotent stem cells could enable the production 

of patient speciic cell types which are fully immuno-compatible with the original donor thereby avoiding the need for immune 

suppressive treatment during cell based therapy. Here, we will present the challenges and the most recent advances toward the use 

of hIPSC lines for cell based therapy of inherited metabolic disorders. 

Daniel Gaudet, MD, PhD 

Gene Replacement Therapy: Lessons from Lipoprotein Lipase Deiciency 

Lipoprotein Lipase Deiciency (LPLD) is a rare autosomal recessive disease characterized by severe hypertriglyceridemia, 

chylomicronemia and risk of recurrent pancreatitis or other complications. AAV1- LPL S447X gene therapy (Glybera ®) has been 

developed to decrease the clinical morbidity of LPLD. The treatment is based on the rationale that by adding episomal copies 

of functional LPL genes into muscle cells lacking active LPL, metabolic function could be improved or restored. The clinical 

development program for Glybera included two observational studies, three open-label interventional studies and one case 

note review analysis. Based on the results of these studies, Glybera gene therapy has recently been approved by the European 

Commission, demonstrating that it is possible for a gene-based medication to be approved despite all the barriers, which include 

the rarity of the disease, the complexity of the treatment as well as a huge amount of technical, clinical, social, economical and 

ethical issues. Several key questions regarding the eficacy, safety, cost-effectiveness, accessibility and impact of Glybera on the 

treatment of LPLD will require time and long-term follow-up to be answered. These questions will be discussed with an emphasis 

on the clinician’s perspective and the clinician-patient relationship. Given the time and cost required to develop gene-based 

treatments, the approval of Glybera could have an encouraging effect on the development of gene therapy for other diseases. 

Alessandro Aiuti, MD, PhD 

Progress in Gene Therapy for Wiskott-Aldrich Syndrome 

Wiskott-Aldrich Syndrome (WAS) is a primary immunodeiciency associated with eczema, thrombocytopenia, and high risk of 

developing autoimmunity and cancer. Gene therapy with ex vivo transduced hematopoietic stem cells (HSC) could represent a valid 

therapeutic option for patients lacking an HLA-identical donor. We designed a phase I/II clinical trial based on reduced intensity 

conditioning and infusion of autologous HSC transduced with a lentiviral vector expressing WAS protein under its endogenous 

promoter. High-throughput vector integration analyses demonstrated highly polyclonal hematopoiesis in vivo and improved safety 

proile over previously used retroviral technology platforms. Gene therapy resulted in unprecedented levels of progenitor cell 

engraftment, restored WAS expression and correction of the disease phenotype in affected children. 



9:45 am - 11:45 am 


microRNA on Cellular Immune Response 

CO-CHAIRS: Shulin Li, PhD and Denise Sabatino, PhD 

SPEAKERS 

Ryan M. O’Connell, PhD 

Micro-managing Inlammation 

MicroRNAs represent a powerful mode of posttranscriptional gene regulation, and have recently been linked to inlammatory 

disease in mammals. We have identiied speciic miRNAs that are expressed in immune cells during inlammatory responses, and 

have determined that they are central regulators of distinct types of inlammation. Our work has also unveiled direct, functionally 

relevant mRNA targets of these miRNAs, providing new insights into the molecular networks that govern inlammatory phenotypes. 

Based upon their emerging importance, miRNAs are now being targeted therapeutically in an effort to mitigate human disease. 


microRNA Regulation of Dendritic Cells 

Plasmacytoid dendritic cells (pDC) are major interferon producing cells which are specialized at sensing the initial stages of viral 

infection. Because of this, pDCs are particularly relevant in the host response to vaccines and gene therapy vectors. I will discuss 

our work which has uncovered evidence of a microRNA that is crucial for proper pDC development and response to pathogen 

associated nucleic acids. 


Immunological Considerations of Therapeutic siRNA 

60 



Judy Lieberman, MD, PhD 

Aptamer-siRNA Chimeras Targeting CD4 Cells Inhibit HIV Transmission 

One attractive strategy for interrupting HIV transmission is a topical microbicide, but the need for application around the time of 

sexual intercourse leads to poor patient compliance. Intravaginal application of CD4 aptamer-siRNA chimeras (CD4-AsiCs) targeting 

the HIV coreceptor CCR5, gag and vif protected humanized mice from sexual transmission. In nondividing cells and tissue, RNAimediated 

gene knockdown lasts for several weeks, providing an opportunity for infrequent dosing not temporally linked to sexual 

intercourse, when compliance is challenging. In follow-up studies, we investigated the durability of gene knockdown and protection, 

protection afforded by CCR5 or HIV gene knockdown on their own, and effectiveness of CD4-AsiCs formulated in a gel in polarized 

human cervicovaginal explants and in humanized mice. These data, showing persistent gene knockdown and sustained protection 

that was improved in a gel formulation, suggest that CD4-AsiCs are a promising approach for developing an HIV microbicide. 


9:45 am - 11:45 am 


Realizing the Potential: Getting to Funding, Getting to Approval and Getting to Market 

CO-CHAIRS: Anthony T. Cheung, PhD and Karen Kozarsky, PhD 

SPEAKERS 


Development of a Commercial Gene Therapy Product 

The market authorization of Glybera by the EMA has given development of gene therapies a signiicant boost. In this presentation 

I will discuss regulatory and inancial challenges associated with the development of (future) gene therapy products, which is 

essential for successful transition of academic projects into commercial products. 


Oncolytic and Immunotherapeutic Vaccinia Viruses for Cancer: Clinical Proof-of-concept with JX-594 

(Pexa-Vec) 

This talk will be an overview of oncolytic and immunotherapeutic vaccinia viruses for cancer, with a focus on clinical proof-ofconcept 

data with JX-594 (Pexa-Vec). Dr. Kirn will review this emerging novel therapeutic class, including the scientiic rationale 

for cancer-selectivity, mechanisms-of-action, and human clinical trial data from Phase 1 and 2 studies with Pexa-Vec. Clinical trial 

data will include pharmacokinetics, pharmacodynamics, tumor biopsy analyses, safety, anti-cancer eficacy and survival. Data from 

a randomized dose-inding Phase 2 trial will be presented, demonstrating a dose-related overall survival beneit of high dose Pexa- 

Vec treatment in patients with advanced liver cancer (Nature Medicine 2013). 


Realizing the Potential: Getting to Approval and Getting to Market; Focus on Orphan Indications 

The successful path to approval and market for new drugs is a series of effective decisions. Basic science, clinical research and 

manufacturing expertise represent some of the major stepping stones on this path, although there are many others. Academic 

scientists, regulators, corporate scientists, regulators, patients and patient advocacy groups all play their roles on the journey. For 

new technologies like cell and gene therapy, the beacon that should guide us throughout this journey should be the demonstration 

of truly transformative beneit to patients. 


Artur Isaev, MD, MBA 

Development, Clinical Trials and Registration of Neovasculgen® Gene Therapeutic Drug 

Presentation will cover the key steps in “Neovasculgen” VEGF165 plasmid gene therapy drug development and commercialization 

starting from choosing a target disease to its marketing, including assessment of key characteristics of the future drug, 

determination of the therapeutic approach to be used and design and performance of preclinical studies. The main focus of 

the talk would be on “Neovasculgen” clinical trials design for chronic lower limb ischemia and their main results, which would 

highlight the necessity of precise deinition of the end points to obtain objective data about safety and eficacy of the drug. Finally, 

the presentation would summarize speciicity of drug development in Russia, current stage of “Neovasculgen” development, and 

perspectives of plasmid gene therapy in general. 

PANEL DISCUSSION 

MODERATOR 

Estuardo Aguilar-Cordova, PhD 


61


PANEL 

Thomas W. Chalberg, Jr., PhD 

Artur Isaev, MD, MBA 





9:45 am - 11:45 am 


Target Non Coding RNAs with Oligonucleotides 

CO-CHAIRS: Kevin V. Morris, PhD and Marco S. Weinberg, PhD 

SPEAKERS 

Claes Wahlestedt, MD, PhD 

RNA-Based Strategies To Upregulate Gene Expression 

Recently, key roles for regulatory long noncoding RNAs (lncRNAs) in the regulation of gene expression have emerged. I will review 

current understanding of the mechanisms of action of lncRNAs and their roles in disease, focusing on recent work in the design 

of inhibitors of the natural antisense transcript (NAT) class of lncRNAs, known as AntagoNAT oligonucleotides, and the issues 

associated with their therapeutic application. 

Christine Esau, PhD 

Targeting microRNAs in Human Disease: miR-33 in the Treatment of Atherosclerosis 

microRNAs are regulators of the genome that can modulate the activity of speciic gene networks. Single stranded oligonucleotides 

can be designed to inhibit the activity of individual microRNAs that are overexpressed in human disease. For example, miR- 

33 is a microRNA that is known to be overexpressed in macrophages that may be involved in the formation of atherosclerotic 

plaques. Inhibition of miR-33 has been shown de-repress known target mRNAs such as ABCA1, increase cholesterol eflux 

from macrophages and regress atherosclerotic lesions in rodent models. Thus targeting microRNAs holds great promise for the 

treatment of human diseases. 


Keith Gagnon, PhD 

Long Noncoding RNAs Link Transcriptional Regulation of Inlammatory Pathway Genes 

Coordinated expression of the eicosanoid signaling pathway genes COX-2 and PLA2G4A are regulated by a network of non-coding 

RNAs and gene looping. The long non-coding RNAs overlapping the COX-2 gene promoter can be targeted by complementary 

endogenous and synthetic small RNAs to elicit activation of both COX-2 and PLA2G4A transcription. This activation requires RNAi 

factors like Argonaute 2 and GW182, recruitment of RNA Polymerase II, and chromatin modifying factors. These indings suggest 

that non-coding RNA has the potential to assemble related genes into functional operons for regulation and small RNAs can control 

this mechanism to synchronously activate transcription. 

Kevin V. Morris, PhD 

Long non-coding RNAs, Epigenetics and Gene Expression 

In human cells some non-coding RNAs have been found to transcriptionally modulate gene expression by targeting epigenetic 

complexes to particular loci. The targeted disruption of these suppressor non-coding RNAs can result in targeted activation of 

gene expression. Examples of this form of gene activation in disease models ranging from cystic ibrosis to HIV to cancer will be 

discussed. 

62 




9:45 am - 11:45 am 

ROOM: 150 ABC 

Partially supported by the Juvenile Diabetes Research Foundation. 

Gene and Cell Therapy for Diabetes 

CO-CHAIRS: Nicola Brunetti-Pierri, MD and Lawrence C. Chan, MD 

SPEAKERS 

H. Teresa Ku, PhD 

Pancreatic Stem and Progenitor Cells 

Adult pancreatic stem and progenitor cells are potential unlimited sources of beta cells, however their existence is highly 

controversial. To address this issue, we report here the establishment of two in vitro pancreatic colony assays that enable 

quantitative measurement of self-renewal and differentiation at a single cell level, which is required to deinitively demonstrate 

progenitor cell activities. We ind that single pancreatic colony-forming cells are capable of self-renewal in the presence of Matrigel 

and a Wnt signaling agonist, and can differentiate into ductal-like cells (in the presence of Matrigel) as well as endocrine and acinar 

lineage cells (in the presence of an unique laminin hydrogel) in culture. These results demonstrate robust pancreatic progenitor cell 

activities in vitro. Our results call for further investigation of progenitor cells in the adult pancreas in vivo, a research activity that 

has largely ceased after a series of recent publications reporting negative indings. 

Lawrence C. Chan, MD 

Gene Therapy to Reverse Autoimmune Type 1 Diabetes 

Type 1 diabetes (T1D) happens when most of the body’s pancreatic beta cells are destroyed by autoimmunity. Therefore, to reverse 

diabetes in T1D patients, we need to [1] re-establish suficient beta cell mass, and [2] neutralize the effects of autoimmunity. Our 

laboratory has developed a strategy that couples gene therapy-mediated induction of new beta cell formation in the liver with an 

immunomodulatory regimen that protects the neo-beta cells from autoimmune destruction. This strategy successfully reverses 

diabetes in an autoimmune model in rodents; the concept of therapeutic hepatic islet induction may also work in nonhuman 

primates. 

Andrea Ballabio, MD 

Rescue of Fat Diet-induced Obesity and Diabetes by TFEB-mediated Regulation of Autophagy and Lipid 

Catabolism 

The lysosomal-autophagic pathway is activated by starvation and plays an important role in both cellular clearance and lipid 

catabolism. However, the transcriptional regulation of this pathway in response to metabolic cues is currently uncharacterized. 

Here we show that the transcription factor EB (TFEB), a master regulator of lysosomal biogenesis and autophagy, is induced by 

starvation through an autoregulatory feedback loop and exerts a global transcriptional control on lipid catabolism via PGC1α and 

PPARα. Thus, during starvation a transcriptional mechanism links the autophagic pathway to cellular energy metabolism. Viral 

delivery of TFEB to the liver rescued obesity, diabetes and the metabolic syndrome in both diet-induced and genetic mouse models 

of obesity, suggesting a novel therapeutic strategy for disorders of lipid metabolism. 


Markus Grompe, MD 

Reprogramming Gall Bladder to make Insulin 


11:45 am - 1:00 pm 


1:00 pm - 3:00 pm 


DNA Vectorology & Gene Targeting 

CO-CHAIRS: Matthew Hirsch, PhD and Carol H. Miao, PhD 



63



1:00 pm - 3:00 pm 


Gene & Cell Therapy of Diabetes, Metabolic and Genetic Diseases I 

CO-CHAIRS: Sihong Song, PhD and Stephanie Cherqui, PhD 



1:00 pm - 3:00 pm 

ROOM: 150 ABC 

Neurological Therapy: Neurodegeneration and Imaging 

CO-CHAIRS: Krystof Bankiewicz, MD, PhD and Brian K. Kaspar, PhD 



1:00 pm - 3:00 pm 


Musculo-skeletal Gene & Cell Therapy II 

CO-CHAIRS: Jacques P. Tremblay, PhD and Annemieke Aartsma-Rus, PhD 




1:00 pm - 3:00 pm 


Cancer - Targeted Gene & Cell Therapy 

CO-CHAIRS: Karen S. Aboody, MD and Janaiah Kota, PhD 



1:00 pm - 3:00 pm 


Gene Therapy for Hematologic Diseases: From Models to Clinical Results 

CO-CHAIRS: Fabio Candotti, MD and Arthur W. Nienhuis, MD 



1:00 pm - 3:00 pm 


Gene Regulation and Animal Models 

CO-CHAIRS: David W. Emery, PhD and David M. Spencer, PhD 


64 



Presidential Symposium 330 

3:15 pm - 5:30 pm 


Presidential Symposium 

CHAIR 

SPEAKER 


David Baltimore, PhD 

Gene-based Anti-Viral Protection 

Standard vaccination development methods have not provided protections against certain infectious agents, notably HIV. However, 

very effective monoclonal antibodies to such agents have been found. We have undertaken to use gene therapy-like methods 

to provide protection against HIV and other agents, using Adenovirus-Associated Virus in a methodology we termed Viral 

ImmunoProphylaxis (VIP). This has proved very effective in humanized mice and is being developed for human trials. 

PRESENTATION OF TOP ABSTRACTS: 

Sylvie Rival-Gervier, PhD 

Abstract 322: Kinetics and Epigenetics of Retroviral Silencing in Mouse Embryonic Stem Cells Deined by 

Deletion of a D4Z4 Barrier Element 

Mahzad Akbarpour, DVM 

Abstract 323: Liver-Directed, Insulin-Based Gene Therapy Protects NOD Mice from Diabetes 

Thomas J. Cradick, PhD 

Abstract 324: On-Target Cleavage and Off-Target Activity of TALEN ‘Nickases’ 

Rimas J. Orentas, PhD 

Abstract 325: Targeting B Cell Precursor Acute Lymphoblastic Leukemia (ALL) with Chimeric Antigen 

Receptors (CARs) Speciic for CD19 or CD22 

Exhibit Hall Open: Networking Reception & Poster Session II 

5:30 pm - 7:30 pm 


AAV Vectors - II 



DNA Vectorology & Gene Targeting I 


Gene & Cell Therapy of Diabetes, Metabolic and Genetic Diseases I 




Neurologic Gene & Cell Therapy I 

(Abstracts 376-391; and Abstract 590 moved from Poster Session III; see pages 115-116) 

Cancer - Immunotherapy II 



65


Cancer-Oncolytic Viruses I 


Hematologic and Immunologic Gene & Cell Therapy I 


Gene and Cell Therapy for Pulmonary Diseases 


Immunologic & Host Responses in Gene & Cell Therapy 


Oligonucleotide & RNAi Therapeutics 


Pluripotent Stem Cells 

(Abstracts 465-476; Abstract 475 Withdrawn from Presentation; see pages 121-122) 

Late Breaking Abstracts II 


New Investigator Town Hall Gathering 

7:30 pm - 8:30 pm 


CO-CHAIRS: Jennifer E. Adair, PhD and Luk H. Vandenberghe, PhD 

SPEAKERS 



The Structural Organization of ASGCT 

The ASGCT is a broad organization that serves many needs of its members, and there are numerous opportunities for young 

investigators to become actively involved in our society. This talk will discuss the overall organization of ASGCT, including our 

committee structures, annual meeting, and awards. 


British Society of Gene and Cell Therapy 

Discussion around the role of BSGCT and the opportunities for interactions for early career researchers. 

Luigi M. Naldini, MD, PhD 

Opportunity for Young Scientists to Contribute to the European Society of Gene and Cell Therapy 

Kenzaburo Tani, MD, PhD 

Current Situation of New Investigator Support Opportunities in JSGT 

JSGT have been supporting new investigators by supplying several educational lectures, awards and research grants. We also have 

open several postdoctoral positions for foreign new investigators. 

Zoe Fonseca-Kelly, PhD 

Strategies for Career Success Beyond Academia 

The role and work of the National Postdoctoral Association will be presented along with data pertaining to postdoctoral issues 

and developments. In addition basic strategies in planning a successful career beyond ones postdoc both within and outside of 

academia will be presented. 

66 



Robert M. Frederickson, PhD 

Molecular Therapy and the ASGCT Publishing Program 

This presentation will provide a brief overview of the Society’s lagship journal, Molecular Therapy, as well as the new online-only, 

open-access sibling journals. It will also present a roadmap of the editorial processes at the journals along with suggestions on how 

to navigate these processes successfully. Finally, we will outline opportunities for new investigators to get involved and contribute 

to the ASGCT publishing program. 

New Investigator Mentoring Event 

8:30 pm - 9:30 pm 



7:30 pm - 9:30 pm 


Cell and Gene Therapy in the Eye, Navigating the Road from the Lab to the Clinic 

Supported by the Foundation Fighting Blindness. 

CHAIR: Stephen M. Rose, PhD 

SPEAKERS 

Stephen M. Rose, PhD 

The Role of the Foundation Fighting Blindness in Bringing Treatments to the Clinic - Funding the “The Valley of 

Death” 

Sue Washer, MBA 

Small Company Experience; Maximizing Sources of Funding and Shots on Goal 


Large Company Experience, Navigating Through “New Technology” Challenge in a Large Organization 

New technological advancements drive development of new therapeutic modalities. Newness implies risk of failure which is 

the norm for scientiic endeavors because out of failure often comes insight that propels ields to new heights of understanding. 

Failure is an accepted ingredient of product development as well – obviously well-managed and early failure is ideal in a corporate 

environment. Layering on of new technologies such as gene and cell therapies complicates the process. Navigation through this 

arena will be discussed. 

Michael Young, PhD 

Retinal Stem Cells: Working with Biotech 

I will present my perspective on collaboration with biotech in the development of a stem cell therapy for retinal degeneration. My 

group has worked with Reneuron, Inc. for almost 8 years now, and we hope to translate our work into a clinical product . I will 

discuss the challenges one encounters when working closely with industry. 



67


Workshop 350 

7:30 pm - 9:30 pm 


International Consortium for Gene and Cell Therapy Workshop 

CHAIR: Jacques P. Tremblay, PhD 

SPEAKERS 

Jacques P. Tremblay, PhD 

Introduction to the Creation of an International Consortium of Gene 

Dr. Tremblay has recently published in collaboration with 50 experts in gene therapy a letter in Molecular Therapy proposing 

the creation of an International Consortium of Gene Therapy to accelerate the development of clinical applications. Indeed the 

recent progress in genomics has permitted to identify the genes responsible for 10,000 hereditary monogenic diseases. Although 

each one of these diseases is rare (they are thus designated as orphan diseases), this whole group of diseases affects 8% of the 

human population and is thus a major cost for the public health systems. On the other hand, gene therapy has recently permitted 

successful treatment of some of these hereditary diseases. In particular, the new AAV serotypes permit to deliver a corrective gene 

more eficiently because human are not pre-immunized to some of the new serotypes. There are also several new tools to correct 

a mutated gene: meganucleases, ZFNs, TALENs and the CRISPR system, or intervene at the level of the mRNA: oligonucleotides for 

exon skipping, siRNA and shRNA. Promising results have been recently obtained in several animal models of hereditary diseases. 

The limiting step to be able to push these discoveries to a clinical application is the high cost of producing the clinical grade vectors. 

The workshop during the annual meeting of the ASGCT is to set the basis of that consortium, deine its goals and elect a direction 

committee. Among the proposed objectives will be to lobby governments for increase funding of this type of research, establishing 

international patient registry, developing more GMP facilities to produce the therapeutic agents, making the regulations more 

uniform in various countries. A large part of the workshop will be devoted to an open discussion so that ALL the participants can 

make their suggestions about the objectives and the methods to reach them. 

Yu-Quan Wei, MD, PhD 

Gene Therapy for Genetic Diseases in China 


Current Status of Clinical Grade AAV Vector Production 


Annemieke Aartsma-Rus, PhD 

The Treat-nmd Paradigm 

The TREAT-NMD Alliance is a network for the neuromuscular ield that provides an infrastructure to facilitate and accelerate 

the clinical development of new therapies. During its EU funded period (2007-2011) the network has collaborated with the 

USA to develop tools such as disease registries, standard of care guidelines, a clinical trial site registry, standardized operating 

procedures for preclinical animal studies, has set up the TREAT-NMD advisory committee on therapeutics (TACT), has maintained 

a biobank with samples from neuromuscular disorder patients and has gathered a wealth of information for the neuromuscular 

ield (see www.treat-nmd.eu). Following the end of the EU funding, the Alliance and its tools have been maintained and the 

network is running with other sources of funding. Current focus is on furthering the biobanks, setting up patient registries for 

additional neuromuscular disorders and in more countries, optimizing the clinical trial site registry, optimizing outcome measures 

for ambulant and non-ambulant patients and generating standards of care for additional diseases and updating existing care 

guidelines. The TREAT-NMD Alliance has also given rise to new EU funded projects such as RD-Connect, Neuromics, Optimistic and 

SKIP-NMD. While for the TREAT-NMD infrastructure the disease area is the connecting factor, it can serve as a paradigm for the gene 

therapy consortium where the therapeutic tool is the connecting factor. 

Philip J. Brooks, PhD 

Activities of the Ofice of Rare Diseases Research, NCATS, NIH, in Facilitating Translational Research in Rare 

Diseases Around the World 

68 


Program Schedule, Saturday, May 18, 2013 

ASGCT Business Meeting 

7:30 am - 8:00 am 

ROOM: 150 ABC 

Plenary Session 400 

8:00 am - 10:00 am 


Outstanding New Investigator Symposium 

CHAIR 

SPEAKERS 



A Sweet Side to AAV Biology 

Adeno-associated viruses (AAV) exploit a spectrum of glycans for cell surface attachment and transduction. Understanding the 

structural basis and functional implications of such virus-host interactions can guide development of recombinant AAV vectors 

for clinical gene therapy applications. This talk will highlight our contributions towards discovery of AAV receptors, structural 

determinants of AAV tissue tropism and new strategies to re-engineer AAV-glycan interactions. 


Targeted-Image Guided RNA (TIGR) Therapies 

The successful application of RNAi therapeutics to humans awaits the development of robust delivery technologies. Cell conjugation 

of siRNAs to cell-speciic ligands provides a viable solution to this problem. Synthetic RNA ligands (aptamers) represent an 

emerging class of pharmaceuticals with great potential for targeted therapeutic/diagnostic applications. Here, we describe 

advancements in aptamer technology for enabling the development of effective image-guided RNA therapies. 



69


Michael A. Lalamme, MD, PhD 

Cardiac Repair with Human Pluripotent Stem Cells 

Human pluripotent stem cells have a number of potential advantages for use in myocardial infarct repair, including tremendous 

capacity for expansion in the undifferentiated state and unambiguous cardiac potential. Our group irst reported what are now 

widely applied methods to eficiently guide the differentiation of human embryonic stem cells (hESCs) and induced pluripotent 

stem cells (hiPSCs) into cardiomyocytes, as well as their further differentiation to speciic cardiac subtypes (e.g. pacemaker or 

ventricular myocytes). In early proof-of-concept studies using a rat infarct model, we showed that the transplantation of hESCderived 

cardiomyocytes results in partial remuscularization of the infarct scar and improved contractile function. More recently, 

we have used a guinea pig infarct model to show that hESC-derived cardiomyocyte grafts can integrate electrically and contract 

synchronously with host muscle; and that these grafts exert an intriguing arrhythmia-suppressive effect. Our ongoing efforts are 

focused on promoting the structural and functional maturation of hESC-derived cardiomyocytes, as well as their electromechanical 

integration in vivo. 


T cell Therapy for Viruses and Cancer 

Severe and fatal viral infections remain common after hematopoietic stem cell transplantation. Adoptive transfer of in vitro 

expanded cytotoxic T lymphocytes speciic for EBV, CMV and Adv antigens can treat infections that are impervious to conventional 

therapies. However, the time taken to prepare patient-speciic products, the limited breadth of viral speciicities that can be 

accommodated in a single line, and the lack of virus-speciic T-cells in cord blood and seronegative donors restricts application. In 

this presentation I will summarize our preclinical efforts to address these limitations and will present our clinical trial results using 

these products. 


10:15 am - 12:15 pm 


Late Breaking Abstracts 

CO-CHAIRS: TO BE DETERMINED 




10:15 am - 12:15 pm 

ROOM: 150 ABC 

Adenovirus and Other DNA Virus Vectors 

CO-CHAIRS: Prem Seth, PhD and Miguel Sena-Esteves, PhD 


70 




10:15 am - 12:15 pm 


Nuclease Mediated Genome Editing 

CO-CHAIRS: Shondra M. Miller, PhD and Andy Wilber, PhD 



10:15 am - 12:15 pm 


Gene & Cell Therapy of Diabetes, Metabolic and Genetic Diseases II 

CO-CHAIRS: Gerald S. Lipshutz, PhD and Nicola Brunetti-Pierri, MD 



10:15 am - 12:15 pm 


Cancer - Immunotherapy II 

CO-CHAIRS: Laurence JN Cooper, MD, PhD and Richard G. Vile, PhD 



10:15 am - 12:15 pm 



CO-CHAIRS: Scott Q. Harper, PhD and M. Corinna Palanca-Wessels, MD, PhD 



12:15 pm - 1:45 pm 


12:15 pm - 1:45 pm 

ROOM: 251 E 

Targeting TTR in the Liver 

SPEAKER 



12:15 pm - 1:45 pm 

MEET AT THE SOUTH ENTRANCE OF THE SALT PALACE CONVENTION CENTER 

Huntsman Cancer Institute Tour 

Boxed lunches will be available at the South Entrance shortly before departure to the Huntsman Cancer Institute. 



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1:45 pm - 3:45 pm 


Clinical Trials 

CO-CHAIRS: Donald B. Kohn, MD and Miguel Sena-Esteves, PhD 

SPEAKERS 


Immunotherapy with Viral Speciic T Cells 

Immunotherapy with antigen-speciic T cells has the potential to reconstitute antiviral activity post transplant without inducing 

GVHD and our group has performed a series of clinical studies showing that virus-speciic cytotoxic T lymphocytes (CTLs) can 

reconstitute immunity and treat viral reactivation and disease. To extend this approach to EBV-associated lymphoma occurring in 

the immunocompetent host expressing Type 2 latency, we have generated CTL lines targeting the subimmunodominant antigens 

LMP1 and LMP2 using antigen presenting cells genetically modiied to overexpress either LMP2 alone or LMP1 and LMP2 by 

transduction with an Ad5f35 vectors. 27 of 29 high-risk and/or multiply relapsed patients who received LMP-CTL as adjuvant 

treatment remain in remission for a median of 2.5 years after CTL while 13 of 21 patients with detectable disease had clinical 

responses. In conclusion, immunotherapy with CTL targeting LMP antigens is well tolerated in patients with EBV+ lymphoma and 

infused LMP-CTL can accumulate at tumor sites and induce complete and sustained clinical responses. 

Roger J. Hajjar, MD 

Gene Therapy for Heart Failure 

Congestive heart failure remains a progressive disease with a desperate need for innovative therapies to reverse the course of 

ventricular dysfunction. One of the key abnormalities in both human and experimental HF is a defect in sarcoplasmic reticulum 

(SR) function, which is responsible for abnormal intracellular Ca2+ handling. Deicient SR Ca2+ uptake during relaxation has been 

identiied in failing hearts from both humans and animal models and has been associated with a decrease in the activity of the SR 

Ca2+-ATPase (SERCA2a). Over the last ten years we have undertaken a program of targeting important calcium cycling proteins 

in experimental models of heart by somatic gene transfer. This has led to the completion of a irst-in-man phase 1 clinical trial 

of gene therapy for heart failure using adeno-associated vector (AAV) type 1 carrying SERCA2a. The safety proile of AAV gene 

therapy along with the positive biological signals obtained from this phase 1 trial has led to the initiation and recent completion of 

a phase 2 trial of AAV1.SERCA2a in NYHA class III/IV patients. In the phase 2 trial, gene transfer of SERCA2a was found to be safe 

and associated with beneit in clinical outcomes, symptoms, functional status, NT-proBNP and cardiac structure. Furthermore, the 

recent successful and safe completion of the CUPID trial along with the start of more recent phase 1 trials usher a new era for gene 

therapy for the treatment of heart failure. 

Andrew M. Davidoff, MD 

AAV-mediated FIX Gene Transfer for Hemophilia B 

We are conducting a phase I/II clinical trial of factor IX gene transfer for severe hemophilia B. In the trial we are using a serotype-8 

pseudotyped self-complementary adeno-associated virus (scAAV) vector expressing a codon-optimized coagulation factor IX (FIX) 

transgene (scAAV2/8-LP1-hFIXco). AAV-mediated FIX activity at 1-6% of normal has been established in all participants with 

follow-up now of between 3 months and 3 years following gene transfer. 


C. Frank Bennett, PhD 

Antisense Oligonucleotide Therapies for the Treatment of Neurodegenerative Diseases 

Antisense oligonucleotides are chemically modiied oligonucleotides that are designed to speciically bind to a targeted RNA 

through Watson-Crick base pairing, modulating the function of the RNA. Because antisense oligonucleotides target RNA, rather 

than protein, they can be used to target a broader range of therapeutic targets than can be approached with conventional small 

molecule or protein based drugs. Neurodegenerative diseases are an attractive area for antisense technology in that there a number 

of diseases in which the genetic basis is known and not easily approachable with small molecule drugs. 


1:45 pm - 3:45 pm 

ROOM: 150 ABC 

Do Effective Gene Therapies and Regenerative Medicine Affect Dilemmas of Resource 

Allocation 

CHAIR: TBD 

72 



SPEAKERS 


Augmentation of Muscle Function Via Gene Transfer-The Good, The Bad and The Ugly 

Emerging gene and stem cell therapies for inherited muscle disorders aim to stop muscle degeneration while increasing muscle 

mass and strength. Interventions that regulate muscle mass are also potentially powerful approaches to slow the loss of strength 

that accompany aging (sarcopenia) and cancer (cachexia). However, any safe method developed to increase muscle mass or 

strength could also be attractive for use by individuals desiring greater strength or prowess, either due to slight build or athletic 

ambition. This lecture will review current approaches for enhancing muscle mass and their potential for use and abuse. 

Tho mas H. Murray, PhD 

Ethics, Gene and Cellular Enhancements in Sport and Beyond 

Athletes will be quick to grasp the possible applications of gene and cell therapies for sarcopenia and other diseases for enhanced 

performance in sport. How should we think about the ethics of enhancement in sport and in other spheres of human life Examples 

from sport and from the practice of medicine demonstrate that understanding the ethics of any particular enhancement begins with 

appreciating the values we seek within that sphere and the meaning of that activity in people’s lives. 

Leonard M. Fleck, PhD 

Successful and Safe Gene Therapy Raises New Quandaries of Health Care Justice 

Our fundamental question is this: Given that any society has only limited resources to meet virtually unlimited health care needs, 

how high a priority, ethically speaking, ought gene therapy have in meeting those needs This leads to two subsidiary questions. (1) 

Who can be expected to beneit the most from emerging gene therapies, mostly younger individuals or mostly older individuals (2) 

What can we reasonably expect in the way of outcomes from various emerging gene therapies, cures or only halfway technologies 

so characteristic of medicine today If we had a form of gene therapy that could substantially address the loss of muscle function in 

Duchenne patients and could also address loss of muscle function in the elderly, would we argue from the perspective of health care 

justice in a world of limited resources with unlimited needs that both sorts of patients had an equal more right to this therapy Or 

would we argue instead that the younger Duchenne patients had the stronger just claim to this therapeutic intervention 


1:45 pm - 3:45 pm 


Exploiting Unique Attributes of Vectors for Design of Vaccines and Immunotherapies 

CHAIR: Eric Poeschla, MD 

SPEAKERS 

Shan Lu, MD, PhD 

DNA Immunization – Proof of the Potential of Gene-based Delivery of Vaccines 

While gene therapy remains a challenge to treat any genetic and acquired illness, the emergence of DNA immunization has provided 

a unique model system to study the in vivo expression of proteins by a gene-based vector and the subsequent biological effects. 

Since its discovery in the early 1990s, the understanding of DNA immunization has grown substantially. New results suggest 

that DNA immunization is extremely effective in priming antigen-speciic B cells, establishing the stage for protective antibody 

responses, which are the critical components for an effective vaccine. More signiicantly, such indings are supported by clinical 

studies in humans, indicating a new era of gene-based vaccines. 

Lukas Flatz, MD 

Single Cell Gene Expression Proiling Reveals Qualitatively Distinct CD8 T cells Elicited by Different Genebased 

Vaccines 

Immunological techniques have dramatically advanced since the discovery of the immune system at the beginning of last 

century. However, to further improve immunotherapies and vaccination we have to better understand the events shaping an 

antigen encountering T cell on single cell base. We therefore used microluidics based single cell real-time PCR to analyze vaccine 

elicited antigen-speciic CD8 T cells of similar magnitude, phenotype and functionality as measured by conventional techniques. 

Remarkably, analysis of single cell gene expression proiles enabled the discrimination of previously unrecognized subsets of 

central and effector memory CD8 T cells elicited by the three different gene-based vaccines. Single cell real-time PCR is a promising 

new technology increasing the resolution of T cell analysis. 



73


Aliasger K. Salem, PhD 

Adjuvants for Vector-Mediated Cancer Immunotherapy 

This presentation will cover the use of adjuvants such as biodegradable particles and oligonucleotides to enhance the magnitude of 

antigen-speciic anti-tumor CD8+ T-cell responses generated from treatment with adenovirus based vaccines. The presentation will 

also discuss the use of strategies to suppress negative regulatory elements within the tumor such as myeloid derived suppressor 

cells (MDSCs) and regulatory T-cells (Treg) to augment the anti-tumor activity generated by treatment with adenovirus based 

vaccines. 

Andrew Geall, PhD 

Non-viral Delivery of Self-amplifying RNA Vaccines 

Novartis has developed the SAM(tm) vaccine platform. The Platform takes advantage of cell-free RNA production from a 

transcription reaction and delivery with a synthetic delivery system. The broad utility of this novel vaccine technology has been 

demonstrated with genes encoding antigens from several pathogens and found to elicit broad and potent protective immune 

responses. Responses are comparable to a viral delivery technology, but without the inherent limitations of viral vectors. 


1:45 pm - 3:45 pm 


Host Cell Responses to Viral Vectors 

CO-CHAIRS: John T. Gray, PhD and Dmitry M. Shayakhmetov, PhD 

SPEAKERS 

Vojo Deretic, PhD 

Autophagy - A Biological Paradigm for Everyone 

Autophagy is a homeostatic process that continuously cleanses the cytosol by removing defunct organelles, protein aggregates, 

microbial invaders, and endogenous sources of inlammation. It also supplies the cell with alternative sources of energy and 

nutrients by auto-digestion of the cytoplasm during starvation. Autophagy is of signiicance in cancer, aging, neurodegeneration, 

metabolic disorders, inlammatory disease such as Crohn’s disease, and infectious diseases. The presentation will cover the 

pathway, its connections to disease, and focus on anti-inlammatory aspects of autophagy. 

Roland W. Herzog, PhD 

Innate Immunity to AAV Vectors 

Rapid sensing of pathogen-associated molecular patterns by the innate immune system is a mechanism to quickly shut down 

a viral infection via inlammatory responses or IFN I signaling. While a major concern for other viral gene transfer vectors, 

innate responses to AAV are very short-lived and weak. Nonetheless, TLR9 sensing of the AAV genome has emerged as a critical 

component of the response, and evidence for IFN I induction, induction of NF-kB and expression of inlammatory cytokines has 

been obtained. Importantly, immune responses to vector and in some cases also the transgene product are impacted by innate 

immunity. 


John C. Bell, PhD 

Innate Immunity and Oncolytic Viruses 

Oncolytic Viruses have been engineered or selected to speciically replicate in and kill tumour cells. This selectivity is often based 

upon aberrant signalling pathways that are characteristic of malignant cells. However, normal cells found within the tumour 

microenvironment are exposed to constitutive and elevated levels of cancer cell derived growth factors leading to a disruption of 

normal which in turn can lead to overstimulation of normal cellular regulatory pathways. We have found that both neo-vascular 

endothelial cells and cancer associated ibroblasts are sensitized to oncolytic virus infection and killing following exposure to 

tumour cell derived growth factors. The molecular and biological consequences of this sensitization will be discussed. 

Dmitry M. Shayakhmetov, PhD 

Sensing of Endosomal Rupture by the Host 

To achieve therapeutic beneits using gene delivery vectors, both viral and non-viral vectors must be able to eficiently 

penetrate through the plasma membrane of target cells and deliver the therapeutic cargo into cytosolic and/or nuclear cellular 

compartments. However, dedicated molecular sensors may exist that detect perturbations of the integrity of the plasma membrane 

and endosomal compartments within the cell. When triggered, these sensors activate innate immune defense mechanisms which 

can limit the eficacy of the therapy. We will review current understanding of the molecular mechanisms that are triggered within 

the cell upon virus entry, and present new data on molecular sensors of adenovirus-mediated endosome rupture. 

74 




1:45 pm - 3:45 pm 


Lessons Learned from the Manufacture and Clinical Translation of Gene and Cell Therapy 

Products 

CO-CHAIRS: Gene Liau, PhD and Gabor Veres, PhD 

SPEAKERS 

Barrie J. Carter, PhD 

Overview on Product and Clinical Translation 

After two decades of development, the irst gene therapy in the western world that was recently approved for marketing in Europe 

was an rAAV, glybera. The progress over these two decades in developing and testing gene therapy products will be reviewed using 

rAAV as the illustrative example. Substantial progress has been made in scaled up production of rAAV vectors, at least ive different 

manufacturing systems have been employed to advance products into clinical testing. The puriication and quality testing of rAAV 

vectors has advanced signiicantly and as more products approach licensing analytics for rAAV will be further reined. Pathways 

for preclincal testing of rAAV for safety, toxicity and biodistribution are established. Importantly, clinical studies with rAAV have 

generated clear evidence that clinical beneits can be gained and have also illuminated some important considerations such as host 

immune responses. 

Harold Petry, PhD 

Lessons from the Glybera ® European Approval Process to Build a Gene Therapy Product Pipeline 

The presentation will cover the development of Glybera ® , a gene therapy product based on the use of recombinant Adenoassociated 

virus for gene delivery, designed for patients with Lipoprotein Lipase Deiciency. The European Medicines Agency’s 

Committee for Medicinal Products for Human Use (CHMP) has approved authorisation of Glybera (alipogene tiparvovec) for 

marketing in the European Union. It is intended to treat lipoprotein lipase (LPL) deiciency in patients with severe or multiple 

pancreatitis attacks, despite dietary fat restrictions. Hurdles which were recognized during the development program towards 

market authorization will be discussed. 


Generation of T Cells that Engraft, Expand and Persist 

Dr. Levine directs the Clinical Cell and Vaccine Production Facility and is an Associate Professor in the Department of Pathology 

and Laboratory Medicine at the University of Pennsylvania. The CVPF develops, manufactures and tests novel cell and gene 

therapies in clinical trials at Penn and collaborating institutions. Critical components for generation of cells with non-homologous 

function include 1) source and isolation of cells, 2) method of activation, 3) method of expansion, 4) characterization and testing. T 

lymphocytes can be activated, cultured, and endowed with novel functions ex vivo. Ex vivo modiied cells can engraft, signiicantly 

expand in vivo by >1000 fold, mediate potent clinical responses, and can survive for years. 

Raymond T. Bartus, PhD 

CERE-120 (AAV-neurturin) for the Treatment of Parkinson’s Disease: Experience from 4 Clinical Trials and 

Human Autopsy Data 

CERE-120 is an AAV serotype 2 vector expressing the gene for human neurturin, a neurotrophic factor with the natural ability 

to protect dying dopaminergic neurons in Parkinson’s patients from cell death and stimulate their ability to produce tyrosine 

hydroxylase (TH), an enzyme necessary dopamine synthesis. Since CERE-120 can inhibit cell death and stimulate dopamine, it has 

the potential ability to provide both symptomatic improvement as well as slow the neurodegenerative process, hence be disease 

modifying. Ceregene has completed over 24 preclinical studies on CERE-120 in multiple animal models of Parkinson’s disease as 

well as multiple preclinical toxicology studies. Ceregene has completed 4 CERE-120 clinical trials including 2 Phase 1 studies and 2 

Phase 2/2b studies in Patients with moderately advanced Parkinson’s disease. Data from these studies as well as from the analysis 

of 4 brains obtained at autopsy from subjects treat with CERE-120 will be discussed. 



75



1:45 pm - 3:45 pm 


Technological Advances in Non-Viral Gene Transfer & Targeting 

CO-CHAIRS: Richard Heller, PhD and Yoichi Negishi, PhD 

SPEAKERS 

Daniel Scherman, PhD 

Improved Eficiency Biosafe Plasmid for Sustained Expression in Various Tissues in Non-Viral Gene Therapy 

Nonviral gene therapy requires plasmid eukaryotic expression vectors that meet deined criteria such as biosafety and low 

production cost. We have designed a novel antibiotic-free selection system, the pFAR plasmids, which is based on the suppression of 

a chromosomal amber mutation in the essential thyA gene by a plasmid-borne function. The electrotransfer of LUC-encoding pFAR 

into muscle led to high luciferase activities, and in skin and transplanted tumors, transgene expression levels were at least one log 

superior and more sustained with pFAR plasmid electrotransfer, as compared with a plasmid carrying a kanamycin resistance gene. 

Most importantly, pFAR plasmids hydrodynamic delivery to liver lead to more than 9 months high and stable transgene expression, 

thus avoiding the classically observed liver gene extinction, by a process whose mechanism will be analyzed. 

Harvinder Singh Gill, PhD 

Microneedles for Gene and Oligonucleotide Delivery 

Tens to hundreds of minimally invasive microneedles conigured in an array-format with controlled spacing can be used to 

uniformly deliver genes and oligonucleotides to tissues for therapeutic and diagnostic applications. This presentation will describe 

our results from the use of microneedle arrays to deliver a gene-based hepatitis C vaccine to the skin in a mouse model, and the 

ability of microneedles to deliver oligonucleotides into cells situated in 3-D tissues. 

Andrew M. Scharenberg, MD 

High Eficiency Genome Editing Using Endonuclease/Exonuclease Co-expression 

An important factor in applying gene editing technologies in translational applications is the eficiency at which a target gene can 

be modiied in the desired cell population. We have explored the use of exonuclease/endonuclease co-expression as a means to 

increase the eficiency of gene editing by several endonuclease platforms, and in both tumor and primary cell types. Results from 

evaluation of several different exonuclease/endonuclease platform combinations, along with mechanistic analysis of how speciic 

exonucleases are interacting with free DNA ends will be presented. 

Evan C. Unger, MD 

Gene Delivery with Ultrasound and Microbubbles 

Safe, highly eficient vectors for gene delivery should expand clinical applications of gene therapy. Ultrasound is widely used for 

diagnostic imaging and has therapeutic applications for heating tissues, increasing tissue and capillary permeability and other 

applications in drug and gene delivery. Microbubbles lower the thresh-hold of energy for cavitation by ultrasound. FDA approved 

ultrasound contrast agents are currently being tested as gene delivery agents, but existing agents probably do not bind the genetic 

materials. New microbubbles that speciically bind genetic materials are also being tested. In my presentation I will review current 

developments in this ield and touch upon opportunities for development of new agents. 


76 



Poster Session III & Reception 

3:45 pm - 5:45 pm 




AAV Vectors - III 


Adenovirus and Other DNA Virus Vectors II 

(Abstracts 552-564; Abstract 561 has been moved to Poster Session I after Abstract 138; see pages 129-130) 

DNA Vectorology & Gene Targeting II 


Gene & Cell Therapy of Diabetes, Metabolic and Genetic Diseases II 

(Abstracts 578-587; Abstract 588 has been withdrawn; see page 131) 

Neurologic Gene & Cell Therapy II 

(Abstracts 589-604; Abstract 590 has been moved to Poster Session II after Abstract 391; see pages 131-132) 



Cancer - Immunotherapy III 


Cancer-Oncolytic Viruses II 


Cancer-Targeted Gene & Cell Therapy: Cell Therapy / Immunotherapy / Cellular Targets 


Hematologic and Immunologic Gene & Cell Therapy II 


Late Breaking Abstracts III 

(Abstracts 733-756; Abstracts 734, 745, and 749 Withdrawn from Presentation; see pages 137-138) 



77

ASGCT 16th Annual Meeting 

May 15-18, 2013 

Salt Palace Convention Center (Hall C/D) 

Salt Lake City, Utah 

Exhibit Hall Floor Plan 

78 


Exhibitor Descriptions 

Aldevron 

3233 15th Street South 

Fargo, ND 58104 

Phone: (701) 306-2675 

Fax: (701) 280-1642 

E-mail: vchambers@aldevron.com 

Website: www.aldevron.com 

Booth Number: 203 

Aldevron’s custom plasmid puriication service is used 

to support many cell and gene therapy applications. Visit 

with our team to learn about new options for bench scale, 

research grade, and clinical grade DNA production. Aldevron 

also provides ready-to-use plasmids including cytokine, 

reporter, and viral helper vectors. We can also produce 

recombinant proteins, mRNA, and genetic antibodies. 

Aldevron’s mission is to advance science and it would be a 

privilege to work with your group. 

AltheaDx 

3550 Dunhill Street 

San Diego, CA 92121 

Phone: (858) 224-7200 

Fax: (858) 224-7220 

E-mail: abudoff@altheadx.com 

Website: www.altheadx.com 


AltheaDx offers comprehensive Biodistribution Studies 

under GLP, designed to determine the distribution and 

persistence of cellular and gene therapy and viral vector 

products at key tissue sites. Our custom designed real-time 

qPCR analysis provides a powerful tool for assessing the 

safety and eficacy of nucleic acid and cellular therapeutics. 

American Society of Gene & Cell Therapy 

555 East Wells Street 

Milwaukee, WI 53202 

Phone: (414) 278-1341 

Fax: (414) 276-3349 

E-mail: info@asgct.org 

Website: www.asgct.org 


Stop by the ASGCT membership booth to ind out where the 

2014 Annual Meeting will be held and to ind out more about 

ASGCT activities. 

Biomiga, Inc. 

10637 Roselle Street 

Suite C 


Phone: (858) 597-0602 

Fax: (858) 538-1698 

E-mail: info@biomiga.com 

Website: www.biomiga.com 


Featured products: 

- Virus Puriication Kits (Adeno virus, Adeno associate virus, 

Retrovirus and Lentivirus) 

- DNA/RNA Puriication Kits (Membrane and Magnetic 

beads). 

Contract services: 

- From gene to virus: gene synthesis, cloning, mutagenesis, 

plasmid puriication, virus production and validation. 

- shRNA design, gene knockdown validation, stable cell line 

generation and single cell cloning. 

BTX/Harvard Apparatus 

84 October Hill Road 

Holliston, MA 01746 

Phone: (800) 272-2775 

Fax: (508) 429-5732 

E-mail: support.btx@harvardapparatus.com 

Website: www. btxonline.com 


BTX Harvard Apparatus is a global manufacturer and 

distributor of electroporation and electrofusion systems 

and accessories. Our product offering accommodates a 

wide range of standard applications along with specialty 

applications such as in vivo electroporation as well as large 

volume transfection and cell fusion. 

CellGenix GmbH 

One New Hampshire Ave., Suite 125 

Portsmouth, NH 03801 

Phone: (603) 373-0408 

Fax: (603) 373-8104 

E-mail: mcdermott@cellgenix.com 

Website: www.cellgenix.com 


CellGenix manufactures high quality cytokines and media 

for ex-vivo cell culture of Dendritic Cells, T-Cells, NK-Cells, 

Hematopoietic stem cells, Chondrocytes and Mesenchymal 

stromal cells. Our products are manufactured in accordance 

with GMP guidelines and USP and are all component 

free providing reliability, safety and reproducible results 

for pre-clincal and clinical applications. CellGenix has built 

strong partnerships with clinical centers world-wide in 

cancer and regenerative medicine. 


79


Charles River 

251 Ballardvale Street 

Wilmington, MA 01887 

Phone: 1-877-criver1 

Fax: (978) 988-9236 

E-mail: askcharlesriver@crl.com 

Website: www.criver.com 


Charles River supports biomedical researchers with 

services spanning the drug development continuum. Our 

comprehensive preclinical offerings include the design, 

execution and documentation of GLP safety tests before 

and after clinical trials begin. These safety programs 

are designed to mitigate complications of cross-species 

reactivity and risk factors inherent to developing novel 

cellular therapeutics. 


Room 5059 

Colket Translational Research Building 

3501 Civic Center Blvd. 

Philadelphia, PA 19147 

Phone: (267) 426-5256 

Fax: (215) 590-3660 

E-mail: sunj@email.chop.edu 

Website: www.research.chop.edu/programs/ccmt/ 


CCMT is dedicated to the understanding, development, and 

application of gene and cell therapies and the promotion of 

professional and public education in the ield. The Center 

fosters a multidisciplinary approach among researchers 

and scientists working to discover new therapies in search 

of cures for debilitating and life threatening childhood 

disorders. 

Cincinnati Children’s Hospital Medical Center 

3333 Burnet Ave. ML 7013 

Cincinnati, OH 45229 

Phone: (513) 803-1066 

Fax: (513) 636-1446 

E-mail: han.vanderloo@cchmc.org 

Website: https://research.cchmc.org/translationalcores/ 


The Cincinnati Children’s Hospital Translational 

Laboratories were established in 2003 to eficiently move 

innovative ideas from the bench to the bedside. We offer a 

one-stop shop approach to support research, scale-up, GMP 

manufacturing, testing, quality assurance, and clinical trial 

support of your novel cell and gene therapies. 

First Biotech Inc. 

340 West State Street 

Athens, OH 45701 

Phone: (614) 600-3341 

Fax: (740) 597-1548 

E-mail: lhallak@irstbiotech.com 

Website: www.FirstBiotech.com 


First Biotech Inc. is a DNA research reagents company. It 

manufactures a high idelity First Mutation site directed 

DNA mutagenesis kit for large plasmids up to 50 kilobase 

pairs. The kit is based on a new patented technology that 

does not require traditional sub-cloning or cloning vectors. 

Florida Biologix 

13702 Innovation Drive 

Alachua, FL 32615 

Phone: (386) 418-8199 

Fax: (386) 462-7838 

E-mail: jiannarelli@cerhb.ul.edu 

Website: www.loridabiologix.ul.com 


Florida Biologix ® is a biologics contract development, 

manufacturing, and testing organization staffed with 65 

industry-experienced personnel in a 35,000 ft 2 

facility 

offering a variety of services, including: cell line engineering, 

cell banking, process development, assay development, 

cGMP vector/protein production in mammalian and insect 

cells, cell therapies, aseptic illing, product testing, and 

regulatory support. 

GE Healthcare 

800 Centennial Avenue 

Piscataway, NJ 08854 

Phone: (800) 526-3593 

Fax: (877) 295-8102 

E-mail: liz.pita@ge.com 

Website: www.gelifesciences.com 


GenScript USA Inc. 

860 Centennial Ave 

Piscataway, NJ 08854 

Phone: (732) 885-9188 

Fax: (732) 210-0262 

E-mail: info@genscript.com 

Website: www.GenScript.com 


Founded in 2002, GenScript is now the world’s leading gene 

synthesis company, and is particularly skillful with long and/ 

or complex sequences. Besides, GenScript offers a variety 

of services and products for preclinical drug discovery. 

Headquartered in USA, GenScript has currently 1,300+ 

employees worldwide with subsidiaries in Europe, Japan and 

China. 

80 



HypOxygen 

5111 Pegasus Ct., Ste. H 

Frederick, MD 21704 

Phone: (877) 497-6994 

Fax: (301) 662-8096 

E-mail: info@hppoxygen.us 

Website: www.hypoxygen.us 


HypOxygen is featuring the HypOxystation hypoxia 

incubator, designed for physiological cell culture research. 

Scientists have precise control of oxygen, carbon dioxide, 

temperature, humidity and can manipulate cells in-situ. We 

will also be showcasing our new INCOmed CO2 incubators, 

which boast Peltier technology, accurate infrared (NDIR) 

CO2 sensors and custom options. 


2425 Ridgecrest Dr. SE 

Albuquerque, NM 87108 

Phone: (505) 348-9534 

Fax: (505) 348-4983 

E-mail: aholley@lrri.org 

Website: www.lrri.org 


The Lovelace Respiratory Research Institute is a private notfor-proit 

biomedical research organization. Lovelace offers 

animal models of disease, evaluation of eficacy of potential 

new therapeutics, and pre-clinical assessments including 

evaluation of pharmacokinetics/biodistribution in large and 

small animal models. Studies are routinely performed under 

GLP guidelines. 

Mary Ann Liebert, Inc. 

140 Huguenot Street 

New Rochelle, NY 10801 

Phone: (914) 740-2100 

Fax: (914) 740-2101 

E-mail: info@liebertpub.com 

Website: www.liebertpub.com 


Mary Ann Liebert Inc. publishes the premier journal 

program in gene therapy, consisting of Human Gene Therapy, 

HGT Methods, & new in 2013, HGT Clinical Development. 

This expanded coverage provides unprecedented access 

to research and applications critical to the evolution of the 

ield! Visit us @ booth# 107. 

Miltenyi Biotec 

2303 Lindbergh Street 

Auburn, CA 95602 

Phone: (530) 887-5385 

Fax: (530) 888-8925 

E-mail: tausha@miltenyibiotec.com 

Website: www.miltenyibiotec.com 


Miltenyi Biotec provides MACS® Technology, the gold 

standard in cell separation for research and clinical scale, 

as well as sample preparation, cell analysis and molecular 

biology products and services to the biomedical research 

community. The company also manufactures cGMP 

custom reagents, antibodies, proteins, peptides, and other 

molecules. 

MPI Research 

54943 North Main Street 

Mattawan, MI 49071 

Phone: (269) 668-3336 

Fax: (269) 668-4151 

E-mail: info@mpiresearch.com 

Website: www.mpiresearch.com 


MPI Research is a preclinical and early clinical CRO that 

provides discovery, surgery, safety evaluation, bioanalytical, 

and analytical services. We exceed expectations 

through consistency and quality, with a commitment 

to communication and innovation, delivering beneits 

throughout all phases of development. Learn how we can go 

beyond for you at www.mpiresearch.com. 

NanoSight 

6660 N. High Street 

Suite 2A 

Worthington, OH 43085 

Phone: (614) 888-0023 

Fax: (614) 987-0045 

E-mail: admin@nanosight.com 

Website: www.nanosight.com 


NanoSight manufactures instruments for Counting, Sizing 

and Visualizing Nanoparticles from 10 nm to 2.0 µm. Size 

& Concentration measurements along with the ability to 

visualize the nanoparticles in real time and a captured video 

of the sample being analyzed are created for each analysis. 


81


National Gene Vector Biorepository (NGVB) 

980 W. Walnut Street R3 C602 

Indianapolis, IN 46202 

Phone: (317) 274-4519 

Fax: (317) 278-3327 

E-mail: lrubin@iupui.edu 

Website: https://www.ngvbcc.org 


The National Gene Vector Biorepository provides support 

for gene therapy research by offering several services. Check 

out the reagent repository comprising cell lines, plasmids, 

vectors and other tools used in gene therapy research. There 

are archiving services to meet FDA requirements. The NGVB 

will store clinical post-trial samples, inal product reserves, 

and master cell banks as well as pharmacology/toxicology 

samples. A pharm/tox database containing gene therapy 

tox studies is a valuable resource. We offer assistance 

with insertional mutagenesis analysis through the SeqMap 

Bioinformatics Tool. Check out the Gene Therapy Data Map a 

tool for locating gene therapy research. Go to https://www. 

ngvbcc.org or come and see us in Booth 412. 

Nature Publishing Group 

75 Varick Street, 9th Floor 

New York, NY 10013-1917 

Phone: (212) 726-9200 

Fax: (212) 689-9711 

E-mail: institutions@nature.com 

Website: www.nature.com 


Nature Publishing Group brings you leading scientiic and 

medical research. The NPG portfolio combines the continued 

excellence of Nature, its associated research and review 

journals, and 50 leading academic and society journals in the 

life, physical and clinical sciences. Visit Booth 402 for free 

sample copies. 

Nature Technology Corporation 

4701 Innovation Drive 

Lincoln, NE 68521 

Phone: (402) 323-6289 

Fax: (402) 323-6292 

E-mail: hodgson@natx.com 

Website: www.natx.com 


Nature Technology Corporation is a biotechnology 

company that provides industry partners with world class 

technology platforms including safe and eficacious nonviral 

vectors (plasmids) and processes for gene therapy 

and DNA vaccination vector production. NTC also provides 

cost effective outsourcing of product development services 

including synthetic gene design, vector construction, plasmid 

DNA manufacturing and recombinant proteins. 

Nexcelom Bioscience 

360 Merrimack Street, Building 9 

Lawrence, MA 01843 

Phone: (978) 327-5340 

Fax: (978) 327-5341 

E-mail: lvavra@nexcelom.com 

Website: www.nexcelom.com 


Nexcelom’s Cellometer line of simple-to-use cell counters 

automate manual cell counting procedures by obtaining 

accurate counts, viability, and cell sizes in less than 30 

seconds & only 20uL of sample. Fluorescence detection 

capabilities enable fast & simple determination of GFP 

transfection rates, PI-viability, & direct counting of WBCs 

without lysing. 

NHLBI Gene Therapy Resource Program 

2 Rockledge Center, 6701 Rockledge Drive 

Bethesda, MD 20892-7940 

Phone: (301) 435-0477 

Fax: (301) 628-1929 

E-mail: gtrpccc@s-3.com 

Website: www.gtrp.org 


The NHLBI Gene Therapy Resource Program (GTRP) 

facilitates the translation of gene therapy research 

into clinical interventions primarily in heart, lung, and 

blood diseases. Services provided by the Program 

include preclinical and clinical-grade vector production, 

pharmacology/toxicology testing, immunology testing, 

assistance with regulatory activities, and partial inancial 

support of clinical trials. 

NIH Office of Biotechnology Activities 

6705 Rockledge Dr. 

Suite 750 

Bethesda, MD 20892-7985 

Phone: (301) 496-9838 

Fax: (301) 496-9839 

E-mail: oba@od.nih.gov 

Website: http://oba.od.nih.gov 


NIH Ofice of Biotechnology Activities staff is available to 

answer questions about human gene transfer research 

under the NIH Guidelines and provide information about 

GeMCRIS, a web-based, information resource and analytical 

tool that provides information about HGT trials and enables 

investigators to ile adverse event and annual reports 

electronically. 

82 



Panasonic Healthcare Company of North America 

1300 Mitchell Drive, Suite A 

Wood Dale, IL 60191 

Phone: (800) 858-8442 

Fax: (630) 238-0074 

E-mail: phc_info@us.panasonic.com 

Website: www.panasonic.com/biomedical 


Panasonic Healthcare Company is a global, leading provider 

of energy, environment, and lifestyle applications with 

laboratory equipment for pharmaceutical, life science and 

biotechnology applications. Product lines include spaceeficient 

VIP ® ultra-low temperature freezers, cryogenic 

freezers, pharmaceutical and medical refrigerators, cell 

culture incubators, plant growth chambers, and portable 

autoclaves. 

Penn Vector Core 

125 South 31st Street, Suite 2000 


Gene Therapy Program 

Perelman School of Medicine 

Philadelphia, PA 19104 

Phone: (215) 573-0633 

Fax: (215) 261-2442 

E-mail: aksandhu@mail.med.upenn.edu 

Website: http://www.med.upenn.edu/gtp/vector_core.shtml 


Penn Vector Core is a state-of-the-art viral vector production 

facility for investigators both within and external to the 

University of Pennsylvania. The main objective of the Core is 

to make various types of high quality viral vectors for gene 

transfer/Vaccine studies at the preclinical and basic research 

level. 

PerkinElmer 

68 Elm Street 

Hopkinton, MA 01748 

Phone: (781) 663-6900 

E-mail: CustomerCareUS@perkinelmer.com 

Website: www.perkinelmer.com 


Biological complexity raises questions requiring 

translational research from the well, to the cell, to the animal 

and back again. PerkinElmer enables you to approach 

your target from multiple perspectives: locate, detect and 

quantitate your biology of interest; analyze and understand 

it in wider physiological contexts. 

PolyPlus-Transfection 

Bioparc BP 90018 

Illkirch SA 67401 

France 

Phone: 33-(0)3-90-40-61-80 

Fax: 33-(0)3-90-40-61-81 

E-mail: info@polyplus-transfection.com 

Website: www.polyplus-transfection.com 


Polyplus transfection provides state-of-the-art quality 

transfection reagents for in vitro and in vivo DNA and 

siRNA delivery. Polyplus also offers qualiied reagents for 

bioproduction including virus production for the clinic and 

supplies GMP grade reagents for human gene therapy and 

cell therapy clinical trials. 

REGENX Biosciences, LLC 

750 17th Street NW 

Suite 1100 

Washington, DC 20006 

Phone: (202) 778-2365 

Fax: (202) 785-7439 

E-mail: jbrown@regenxbio.com 

Website: www.regenxbio.com 


REGENX BioSciences is leading the effort to translate 

promising gene delivery applications into a pipeline of 

next generation personalized therapies for a range of 

severe diseases with serious unmet needs. We believe that 

the NAV technology, to which we have exclusive rights, 

represents the potential promise of curing the root cause of 

disease rather than the symptoms, and we are committed to 

establishing best in class standards for our NAV vectors. 

SAFC 

BioReliance by SAFC 

Glasgow G20 0XA 

UNITED KINGDOM 

Phone: +44(0)141 576 2462 

Fax: +44(0)141 948 0000 

E-mail: susan.livingston@bioreliance.com 

Website: www.safcglobal.com 



83



262 Danny Thomas Place 

Memphis, TN 38015 

Phone: (901) 595-2750 

Fax: (901) 595-5376 

E-mail: postdoc@stjude.org 

Website: www.stjude.org/research 


At St Jude Children ’s Research Hospital, a non-proit 

biomedical research institution in Memphis, TN, 190 basic 

science and clinical researchers investigate the molecular 

basis of normal cellular and diseased processes. Visit our 

booth to discuss and apply for Postdoctoral Fellowship 

positions. 

Contact Information: 


Ofice of Academic Programs 

262 Danny Thomas Place, MS 276 

Memphis, TN 38105 

Email: postdoc@stjude.org 

http://www.stjude.org/research 

The Baker Company 

P. O. Drawer E 

Sanford, ME 04073 

Phone: (800) 992-2537 

Fax: (207) 324-3869 

E-mail: bakerco@bakerco.com 

Website: www.bakerco.com 

Booth Number: 302 & 304 

For 60 years, The Baker Company has delivered high quality 

contamination control products to meet the unique and 

evolving needs of laboratory professionals. Learn about 

our new generation of high-performance, precision-crafted 

biomedical refrigeration, controlled atmosphere and 

Ruskinn hypoxia workstations on our website at www. 

bakerco.com. 

TriLink BioTechnologies, Inc. 

9955 Mesa Rim Road 


Phone: (858) 546-0004 

Fax: (858) 546-0020 

E-mail: info@trilinkbiotech.com 

Website: www.trilinkbiotech.com 


TriLink manufactures custom oligonucleotides, modiied 

nucleoside triphosphates and CleanAmp PCR products 

for the diagnostic and OEM markets. In addition, custom 

chemistry, long RNA transcript synthesis and contract 

research services and ISO/QSR compliant cGMP production 

facilities are offered. TriLink’s solutions help advance drug 

discovery and biomedical research. 

UAB Vector Production Facility 

550 11th Street South 

Birmingham, AL 35294-4558 

Phone: (205) 975-9871 

Fax: (205) 934-9511 

E-mail: tvstrong@uab.edu 

Website: www.uab.edu/vector/ 


The goal of the UAB Vector Production Facility (VPF) is to 

accelerate the translation of novel biological therapies into 

Phase I human clinical trials. The UAB VPF produces largescale, 

high quality viral vectors, cell banks and recombinant 

proteins for research and clinical applications, in accordance 

with current Good Manufacturing Practices. 

UNC Gene Therapy Vector Core 

7119 Thurston Bowles 

Chapel Hill, NC 27599 

Phone: (919) 962-3285 

Fax: (919) 843-9410 

E-mail: vectorcore@unc.edu 

Website: http://genetherapy.unc.edu/jvl.htm 


University of Iowa/Gene Transfer Vector Core 

Gene Transfer Vector Core 

500 Newton Road 

221 EMRB 

Iowa City, IA 52242 

Phone: (319) 335-6726 

Fax: (319) 335-6895 

E-mail: maria-scheel@uiowa.edu 

Website: www.uiowa.edu/~gene/ 


The GTVC’s objective is to support investigators in the 

use of gene transfer technologies. The Core utilizes 

molecular biology techniques to engineer and develop viral 

vectors based on multiple vector systems. Core staff and 

investigators work together allowing for cross fertilization 

of ideas, technical advancements, and innovations in vector 

design. 

84 



University of Utah Blood & Marrow Transplant 

Program - Cell Therapy & Regenerative Medicine 

Facility 

675 Arapeen Dr., Suite 300 

Salt Lake City, UT 84108 

Phone: (801) 585-6262 

Fax: (801) 585-3222 

E-mail: joanna.reems@hsc.utah.edu 

Website: http://medicine.utah.edu/cell/ 


The Cell Therapy and Regenerative Medicine Program at 

the University of Utah supports Hematopoietic stem cell 

transplants and delivers innovative cellular and tissue 

engineered products to patients by providing comprehensive 

“bench to bedside” services that coordinate the efforts of 

clinicians, researchers, and bioengineers. 

VGXI, Inc. 

2700 Research Forest Drive 

Suite 180 

The Woodlands, TX 77381 

Phone: (281) 296-7300 

Fax: (281) 296-7333 

E-mail: info@vgxii.com 

Website: www.vgxii.com 


VGXI Inc. is a leading provider of DNA plasmid 

manufacturing and development services for DNA vaccine 

and gene therapy clinical research. The Company has an 

outstanding track record of success manufacturing plasmid 

products under GMP for clients in the US, Europe, and 

Australia. 

Waisman Biomanufacturing 

1500 Highland Avenue 

Madison, WI 53705 

Phone: (608) 263-8940 

E-mail: info@gmpbio.org 

Website: www.gmpbio.org 


Waisman Biomanufacturing has in-depth experience 

manufacturing a wide range of biotherapeutics for human 

Phase I/II clinical trials including gene therapeutics, cell 

therapeutics, vaccines, and recombinant proteins. Services 

include process development through cGMP production 

and ill/inish capabilities— all with full quality system 

support—and comprehensive support for your IND iling. 

Wilson Wolf Corporation 

33 5th Avenue, NW 

New Brighton, MN 55112 

Phone: (651) 628-9259 

Fax: (651) 628-9259 

E-mail: info@wilsonwolf.com 

Website: www.wilsonwolf.com 


Wilson Wolf created “G-Rex” technology to simplify and 

reduce the cost cell production for Adoptive Cell Therapy. 

Cell production in G-Rex devices saves material and labor 

while simultaneously shortening production time, allowing 

the most practical and cost effective process for research and 

clinical work. Stop by our booth for details. 


85

Exhibitor Information 

General Information 

The Exhibit Hall is located in Hall C/D of the Salt Palace 

Convention Center. All participants are urged to allow 

adequate time to visit the exhibits. The companies, 

organizations and institutions exhibiting at the ASGCT 

16th Annual Meeting will be providing you with the latest 

information on products and services directly related to 

your professional needs, as well as recruiting for open 

positions. 

Exhibit Dates 

Exhibits will be open Wednesday, May 15 through Friday, 

May 17, 2013. 

Exhibitor Registration 

The Exhibitor Registration Desk is located at the East 

Registration Area – by the East Entrance on the 2nd loor of 

the Salt Palace Convention Center 

Exhibitor Registration Desk Hours 





7:30 am – 7:30 pm 

Exhibit Move In 


Exhibit Hall Hours 


Thursday, May 16 9:35 am – 10:30 am 



9:00 am – 9:45 am 


5:30 pm – 7:30 pm 

Exhibit Move Out 


7:30 pm – 9:30 pm 

Note: All exhibit booths must be staffed during hall hours. 

Packing of equipment/literature and the dismantling of 

exhibits will not be permitted until after 7:30 pm on Friday, 

May 17, 2013. 

Exhibitor Badge Policy 

Admission to the Exhibit Hall will be by name badge only. 

Security guards will monitor exhibit hall entrances for 

proper I.D. Exhibit booth personnel must show an oficial 

ASGCT exhibitor name badge in order to gain access to the 

exhibit hall during installation, show, or dismantlement 

hours. Independent contractor personnel, hired by 

an exhibitor to install and dismantle their display, do 

not need to show exhibitor badges, but should possess 

identiication that they are employed by the independent 

contractor. If independent contractors do not possess proper 

identiication, they should obtain a name badge at the onsite 

exhibitor registration desk. 

Endorsement Disclaimer 

Products and services displayed in the Exhibit Hall or 

advertised in this program occur by contractual business 

arrangements between the ASGCT and participating 

companies and organizations. These arrangements do not 

constitute or imply an endorsement by the ASGCT of these 

products and services. 

86 


Abstract Directory, Wednesday, May 15, 2013 


AAV Vector Development & Application 

3:15 PM - 5:15 PM 

Room: Ballroom A 

1 3:15 PM 

Identiication of Novel Vectors Capable of CNS 

Transduction in Adult Mice after Single Round 

Selection Using DNA Shufled AAV Capsid Library 

Sourav Choudhury, Stacy Maitland, Jennifer Ferreira, Miguel 

Sena-Esteves. 

2 3:30 PM 

Re-Engineering Capsid-Glycan Interactions Expands the 

CNS Transduction Proile of AAV Serotype 4 

Giridhar Murlidharan, Laura Sommerville, Troy Ghashghaei, 

Aravind Asokan. 

3 3:45 PM 

Directed Evolution of Adeno-Associated Virus for 

Enhanced Gene Delivery to Neural Stem Cells In Vitro 

and In Vivo 

David V. Schaffer, Melissa A. Bartel, Jae-Hyung Jang, Tandis Vazin. 

4 4:00 PM 

A Rationally Engineered Novel Capsid Variant of AAV9 

for Pripheral Tissue-Detargeted and CNS-Directed 

Systemic Gene Delivery 

Li Zhong, Shaoyong Li, Kim Van Vliet, Mengxin Li, Jun Xie, Jia 

Li, Qin Su, Ran He, Yu Zhang, Huapeng Li, Dan Wang, Jason 

Goetzmann, Terence R. Flotte, Mavis Agbandje-McKenna, 

Guangping Gao. 

5 4:15 PM 

In Vivo Directed Evolution of a Novel Adeno-Associated 

Virus for Therapeutic Outer Retinal Gene Delivery from 

the Vitreous 

Leah C. Byrne, Deniz Dalkara, Ryan R. Klimczak, Meike Visel, Lu 

Yin, William H. Merigan, John G. Flannery, David V. Schaffer. 

6 4:30 PM 

Adeno-Associated Virus Capsid Motif That Inluences 

Tissue Speciic Vector Transduction In Vivo 

Jayme K. Warischalk, Richard J. Samulski. 

7 4:45 PM 

A Species Restricted rAAV Vector Obtained from Viral 

Capsid Shufling 

Leszek Lisowski, Kirk Chu, Mark A. Kay. 

8 5:00 PM 

Solving the T-Cell Problem: Generation of Immune 

Stealth AAV 

Thierry VandenDriessche, Frederico Mingozzi, Nisha Nair, Jessica 

Willems, Hanneke Evens, Omid Ghandeharian, Kathy High, 

Marinee K. Chuah. 


Cancer Immunotherapy I 

3:15 PM - 5:15 PM 

Room: Ballroom B 

9 3:15 PM 

Enhanced DNA Vaccination Against a Tyrosinase (Tyr) 

Mediates Protection and Targets Myeloid-Derived 

Suppressor Cells in a Syngeneic Murine Model 

Taron C. Gorham, Lavanya Mahadevan, Colleen Lucke, Randolph 

Lyde, Kenneth E. Ugen, Niranjan Y. Sardesai, Joseph J. Kim, 

Karuppiah Muthumani, David B. Weiner. 

10 3:30 PM 

First-in-Human Application of Sleeping Beauty System 

for Gene Therapy 

Partow Kebriaei, Helen Huls, Harjeet Singh, Simon Olivares, 

Matthew J. Figliola, Margaret Dawson, Bipulendu Jena, Rineka 

Jackson, Doyle Bosque, Ian McNiece, Gabriela Rondon, Perry B. 

Hackett, Elizabeth J. Shpall, Richard E. Champlin, Laurence J. N. 

Cooper. 

11 3:45 PM 

AAV-Mediated CNS Gene Transfer of Bevacizumab 

Reduces Human Glioblastoma Growth and Increases 

Survival in Mice 

Martin J. Hicks, Kosuke Funato, Lan Wang, Eric Aronowitz, 

Jonathan P. Dyke, Douglas J. Ballon, Vivianne S. Tabar, David F. 

Havlicek, Esther Z. Frenk, Bishnu P. De, Maria J. Chiuchiolo, Dolan 

Sondhi, Neil R. Hackett, Stephen M. Kaminsky, Ronald G. Crystal. 

12 4:00 PM 

NKT Cells as a Novel Platform for Cancer 

Immunotherapy with Chimeric Antigen Receptors 

Andras Heczey, Daofeng Liu, Amy Courtney, Ekaterina Marinova, 

Gengwen Tian, Eric Yvon, Jie Wei, Gianpietro Dotti, Leonid 

Metelitsa. 

13 4:15 PM 

Removal of Physical Barriers inside the Tumor 

Improves T-Cell Therapy 

Hua Cao, Ines Beyer, Vy Phan-Lai, Nora Disis, Andre Lieber. 

14 4:30 PM 

Administration of GD2 Chimeric Antigen 

Receptor Modiied, Tri-Virus Speciic Cytotoxic T 

Lymphocytes after HLA Mismatched Allogeneic 

Stem Cell Transplantation for Relapsed, Refractory 

Neuroblastoma 

Doug Myers, Jiali Sun, Cliona M. Rooney, Natalia Lapteva, Adrian 

P. Gee, Gianpietro Dotti, Bambi Grilley, Malcolm K. Brenner, Robin 

Ryan. 

15 4:45 PM 

Nonclinical and Phase I Clinical Studies with a 

Regulated Adenoviral Gene Delivery of IL-12 Shows 

Promising Clinical Activity in Unresectable Stage III/IV 

Melanoma 

A. Vergara-Silva, J. Barrett, A. Marsh, R. Khosravi-Far, L. A. Martell, 

O. Hamid, T. Chan, T. D. Reed, H. Youssouian, J. Nemunaitis. 



87



Oral Abstract Session 131, continued 


3:15 PM - 5:15 PM 


16 5:00 PM 

Effective and Safe Gene-Modiied T Cells Expressing 

High Afinity NYESO-1 Speciic TCR Variants with 

Silencing Endogenous TCRs 

Sachiko Okamoto, Yasunori Amaishi, Hiroaki Ikeda, Hiroshi Shiku, 

Junichi Mineno. 


Cancer-Oncolytic Viruses 

3:15 PM - 5:15 PM 


17 3:15 PM 

Intraperitoneal (IP) Administration of an Oncolytic 

Measles Virus (MV) Strain Expressing the Sodium 

Iodine Symporter Gene in Patients (pts) with Advanced 

Ovarian Cancer (OvCa) 

Evanthia Galanis, Pamela Atherton, Sean Dowdy, William Cliby, 

Paul Haluska, Jr, Harry Long, Ileana Aderca, Keith Knutson, 

Matthew Block, Mark Federspiel, Stephen Russell, Kah Whye Peng, 

Lynn Hartmann. 

18 3:30 PM 

In Vivo Expansion of a Recipient Population of Cell 

Carriers Allows for Highly Effective Systemic Delivery of 

Oncolytic Reovirus Even in the Presence of Neutralizing 

Antibody 

Timothy Kottke, Oliver Donnelly, Elizabeth Ilett, Jill Thompson, 

Rosa Diaz, Matt Coffey, Peter Selby, Hardev Pandha, Kevin 

Harrington, Alan Melcher, Richard Vile. 

19 3:45 PM 

Baseline Neutralizing Antibody Status Does Not Affect 

Intravenous Delivery of Oncolytic Vaccinia Pexa-Vec (JX- 

594) in Liver Cancer Patients 

Caroline J. Breitbach, Jeong Heo, Mong Cho, Anne Moon, Chang 

Won Kim, James Burke, Theresa Hickman, Kara Dubois, Yeon 

Sook Lee, Manijeh Daneshmand, John C. Bell, Richard Patt, Tae-Ho 

Hwang, David H. Kirn. 

20 4:00 PM 

Induction of Antiviral Genes by the Tumor 

Microenvironment Confers Resistance to Virotherapy 

Yu-Ping Liu, Lukkana Suksanpaisan, Stephen J. Russell, Kah-Whye 

Peng. 

21 4:15 PM 

Superior Antitumoral Activity Induced by a Novel 

Oncolytic Adenovirus Armed with an EGFR-Targeted 

ImmunoRNase 

Ines Fernandez-Ulibarri, Michaela A. E. Arndt, Dominik E. Dorer, 

Sarah Engelhardt, Jürgen Krauss, Dirk M. Nettelbeck. 

22 4:30 PM 

The Oncolytic and Immunotherapeutic Vaccinia 

Virus Pexa-Vec (JX-594) Induces Antibody-Mediated 

Complement-Dependent Cancer Cell Cytolysis 

Mi Kyung Kim, Caroline H. Breitbach, Anne Moon, Jeong Heo, Yu 

Kyung Lee, Mong Cho, Jun Woo Lee, Seung Geun Kim, Dae Hwang 

Kang, Byung Ho Park, John C. Bell, David H. Kirn, Tae-Ho Hwang. 

23 4:45 PM 

Modulation of Innate Immunity with the Anti-CTLA4 

Antibody Ipilimumab (Ipi) in Measles Virotherapy for 

Glioblastoma 

Jayson J. Hardcastle, Hirosha Geekiyanage, Evidio Domingo 

Musibay, Cory Allen, Jin Fang, Aaron Johnson, Evanthia Galanis. 

24 5:00 PM 

Human Bone Marrow-Derived Mesenchymal 

Stromal Cells Permit Delivery, and Enhance Eficacy 

of Systemically Administered Oncolytic Measles 

Virotherapy to Disseminated Human Pre-B ALL Murine 

Xenograft Models in the Presence of Humoral Immunity 

Anna Castleton, Aditi Dey, Bella Patel, Brendan Beaton, Anne 

Aucher, Daniel Davis, Adele Fielding. 


Advancing the Science of Gene Therapy for 

Hematologic Diseases 

3:15 PM - 5:15 PM 

Room: Ballroom C 

25 3:15 PM 

Improved Chemotherapy Eficacy after MGMT(P140K) 

Hematopoietic Stem Cell Gene Therapy in Poor- 

Prognosis Glioblastoma by Patient-Speciic 

Mathematical Modeling 

Jennifer E. Adair, Brian C. Beard, Sandra K. Johnston, Maciej M. 

Mrugala, Russell C. Rockne, Kristin R. Swanson, Hans-Peter Kiem. 

26 3:30 PM 

Therapeutic Levels of Anti-Sickling Hemoglobin 

Production in Erythroid Progeny of Sickle Cell Disease 

CD34+ Cells Following Lentivirus Vector-Mediated Gene 

Delivery 

Fabrizia Urbinati, Phillip W. Hargrove, Sabine Geiger, Zulema 

Romero, Jennifer Wherley, Michael L. Kaufman, Roger P. Hollis, 

Christopher B. Chambers, Derek A. Persons, Donald B. Kohn, 

Andrew Wilber. 

27 3:45 PM 

Superior Stem Cell Mobilization by a Combination of 

Plerixafor+G-CSF in Patients with Thalassemia 

Evangelia Yannaki, Achilles Anagnostopoulos, Garyfalia 

Karponi, Fani Zervou, Varnavas Constantinou, Nikoleta Psatha, 

Asimina Bouinta, Erica Jonlin, Thalia Papayannopoulou, George 

Stamatoyannopoulos. 

88 




Advancing the Science of Gene Therapy for 

Hematologic Diseases 

3:15 PM - 5:15 PM 


28 4:00 PM 

Tracking Fate and Plasticity of T Cells in Humans by 

Retroviral Tagging 

Luca Biasco, Serena Scala, Cristina Baricordi, Nicoletta Cieri, 

Andrea Calabria, Eugenio Montini, Chiara Bonini, Alessandro Aiuti. 

29 4:15 PM 

Quantitative Tracking of the Output of Individual 

Hematopoietic Stem/Progenitor Cells in a Non-Human 

Primate Model 

Chuanfeng Wu, Brian Li, Rong Lu, Alexander Jares, Robert 

Donahue, Irving Weissman, Cynthia Dunbar. 

30 4:30 PM 

Evaluation of Transduced CD34+ Cells To Predict Gene 

Marking Levels In Vivo in Rhesus Gene Therapy Model 

Naoya Uchida, Molly Evans, Aylin Bonifacino, Sandra Price, Allen 

Krouse, Mark Metzge, Matthew Hsieh, Robert Donahue, John 

Tisdale. 

31 4:45 PM 

Unimolecular Microchimerism Achieved through 

Hematopoietic Stem Cell Transplantation Gene Therapy 

Facilitates Adjunctive Therapy with Genetically- 

Modiied Mesenchymal Stem Cells in Hemophilia A 

Allison M. Abuin, H. Trent Spencer, Christopher B. Doering. 

32 5:00 PM 

Rapamycin Treatment of Hematopoietic Stem Cells 

Enhances Lentiviral Transduction through Autophagy 

Xueqian Wang**, Blythe D. Sather**, Iram Khan, Swati Singha, 

Gabrielle Curinga, David J. Rawlings, Bruce Torbett. 


Neurological Therapy: Monogeneic Disorders 

3:15 PM - 5:15 PM 

Room: Ballroom D 

33 3:15 PM 

Intravenous AAV8-MTM1 Prolongs Life and 

Ameliorates Severe Muscle Pathology in Mouse and Dog 

Models of X-Linked Myotubular Myopathy 

Martin K. Childers, Romain Joubert, Melanie N. Holder, Robert 

W. Grange, Jon Doering, Michael W. Lawlor, Christelle Moal, 

Thibaud Jamet, Nathalie Daniele, Samia Martin, Christel Riviere, 

Kimberly Poppante, Thomas Soker, Caroline Hammer, Laetitia Van 

Wittenberghe, Melissa Goddard, Erin Mitchell, Jane Barber, Mark 

E. Furth, Alban Vignaud, Carole Masurier, Philippe Moullier, Alan 

H. Beggs, Alan H. Beggs, Anna Buj-Bello. 

34 3:30 PM 

Augmenting CNS Glucocerebrosidase Activity as a 

Therapeutic Strategy for Parkinsonism and Other 

Gaucher-Related Synucleinopathies 

Pablo S. Sardi, Lamya S. Shihabuddin, Richard L. Sidman, Seng H. 

Cheng. 

35 3:45 PM 

Eficacious and Sustained Canavan Gene Therapy by 

Single rAAV Injections Via IV at Postnatal Day 21 or ICV 

at a 100 Fold Lower Dose 

Seemin S. Ahmed, Reuben Matalon, Guangping Gao. 

36 4:00 PM 

Dramatic Phenotypic Improvement after Adeno- 

Associated Virus Gene Therapy in a Feline Model of 

Sandhoff Disease 

Victoria J. McCurdy, Heather L. Gray-Edwards, Ashley N. Randle, 

Allison M. Bradbury, Aime K. Johnson, Patricia M. Beadlescomb, 

Nancy Morrison, Misako Hwang, Miguel Sena-Esteves, Douglas R. 

Martin. 

37 4:15 PM 

Preclinical Safety and Eficacy of Human CD34+ Cells 

Transduced with Lentiviral Vector for the Treatment of 

Adrenoleukodystrophy 

Lauryn Christiansen, Maria Denaro, John Pierciey, Anne Moser, 

Cynthia Bartholoma, Michael Rothe, Robert Kutner, Christopher 

Baum, Byoung Ryu, Manfred Schmidt, Garrett Heffner, Mitchell 

Finer, Gabor Veres. 

38 4:30 PM 

Preclinical Evaluation of Systemic Delivery of Blood- 

Brain Barrier-Targeted IDUA in Treating CNS Deicits of 

Mice with Hurler Syndrome after Long Term Stem Cell 

Gene Therapy 

Salim S. El-Amouri, Mei Dai, Dao Pan. 

39 4:45 PM 

Broad Correction of Molecular Impairments by a 

Single Systemic rAAVrh74-hSGSH Gene Delivery in a 

Mouse Model of MPS IIIA: Links between the Brain and 

Peripheral Blood 

F. Jason Duncan, Bartholomew J. Naughton, Kimberly Zaraspe, 

Darren Murrey, Tierra Ware, Aaron Meadows, Haiyan Fu, Douglas 

McCarty. 

40 5:00 PM 

Amelioration of Neurologic Disease after AAV-Gene 

Therapy in a Sheep Model of Tay-Sachs Disease 

H. L. Gray-Edwards, S. M. Hubbard, A. E. Randle, M. Hwang, A. M. 

Bradbury, V. J. McCurdy, D. U. Wilson, J. A. Hudson, B. K. Whitlock, 

J. L. Sartin, N. Salibi, R. J. Beyers, T. S. Denney, J. A. Johnson, K. P. 

Riddell, N. R. Cox, M. Sena-Estevez, D. R. Martin. 



89





3:15 PM - 4:15 PM 

Room: 151 ABCG 

41 3:15 PM 

Optimized Polyplexes That Avoid Liver Uptake and 

Maintain Eficient Gene Transfer In Vivo 

Sanjib Khargharia, Koby Kizzire, Mark E. Ericson, Nick J. 

Baumhover, Kevin G. Rice. 

42 3:30 PM 

Synergistic Cancer Therapy by Simultaneous 

Expression and Silencing of Pro-Apoptotic and Pro- 

Survival Genes Using Chimeric Nanoparticles 

Soo Kyung Cho, Jane Kim, Young Jik Kwon. 

43 3:45 PM 

Examining Cell-Penetrating Homochiral Cyclic Peptides 

as siRNA Non-Viral Transfection Agents 

Christiaan D. King, Ria L. Swanekamp, Bradley L. Nilsson, David A. 

Dean. 

44 4:00 PM 

Structure and Silencing Activity of Imidazole- 

Containing Peptide siRNA Polyplexes Are Dependent on 

Hydrogen Bonding 

Szu-Ting Chou, Kellie Hom, Lucas Tricoli, Jason Hustedt, Amy 

Lee, Joonil Seog, Jason Kahn, Qixin Leng, Daoning Zhang, Michael 

Shapiro, Archibald J. Mixson. 



3:15 PM - 4:15 PM 

Room: 150 ABC 

45 3:15 PM 

Inducing Humoral and Cellular Responses to Multiple 

Sporozoite and Liver-Stage Malaria Antigens Using 

pDNA 

Bernadette Ferraro, Kendra T. Talbot, Amritha Balakrishnan, 

Matthew P Morrow, Natalie A. Hutnick, Devin J. Myles, Devon J. 

Shedlock, Nyamekye Obeng-Adjei, Jian Yan, Rachel Shiver, Amir 

S. Khan, Maria Yang, Ami S. Brown, Ulrike Wille-Reece, Ashley 

Birkett, Niranjan Y. Sardesai, David B. Weiner. 

46 3:30 PM 

T-Cells Edited To Express the C34 Peptide from gp41 

Heptad Repeat-2 Fused to CCR5 or CXCR4 Exhibit 

Robust Protection from Diverse HIV-1 Isolates 

Jianbin Wang, George J. Leslie, Jennifer Duong, Kevin L. Hua, Jenny 

J. Yan, Beth Haggarty, Andrea P. Jordon, Josephine Romano, Lei 

Zhang, Edward J. Rebar, Philip D. Gregory, James A. Hoxie, Michael 

C. Holmes. 

47 3:45 PM 

Integrase-Defective Lentiviral Vector-Induced Dendritic 

Cells: Clinical Development and Validation of CD4+/ 

CD8+ T Cell Responses for Combination with Adoptive 

CMV-Speciic T-Cell Therapy 

Anusara Daenthanasanmak, Constanca Figueiredo, Eliana 

Ruggiero, Manfred Schmidt, Arnold Ganser, Renata Stripecke. 

48 4:00 PM 

Engineering Resistance to HIV-1 Infection with a Dual 

Therapeutic Lentiviral Vector 

Bryan P. Burke, Maureen P. Boyd, Michelle L. Millington, Helen 

Impey, Jane Zhang, Maria V. Carroll, Joanna Camba-Colon, Naomi 

Keech, Frederic Delebecque, Orit Wolstein, Annett Howe, Rachel 

Koldej, Tamara Nicolson, Bernard R. Levin, Valerie Rezek, 

Dimitrios N. Vatakis, Scott G. Kitchen, Louis R. Breton, Jeffrey S. 

Bartlett, Geoff P. Symonds. 



4:20 PM - 5:20 PM 


49 4:20 PM 

gc Cytokines IL7 and IL15 Expanded Chimeric Antigen 

Receptor-Redirected T Cells (CAR-T) with Superior 

Antitumor Activity In Vivo 

Yang Xu, Ming Zhang, Aruna Mahendravada, Barbara Savoldo, 

Gianpietro Dotti. 

50 4:35 PM 

Large-Scale Clinical-Grade Retroviral Vector Production 

in iCELLis Nano Bioreactor 

Xiuyan Wang, Malgorzata Olzzewska, Jinrong Qu, Teresa 

Wasielewska, Shirley Bartido, Gregory Hermetet, Michel Sadelain, 

Isabelle Riviere. 

51 4:50 PM 

Large Scale Production of Lentiviral Vectors Using 

Serum Free Suspension Cell Culture System 

Amelia H. Thomas, Kevin Brown, Francis H. Pierciey, Jr, Gabor 

Veres, Byoung Y. Ryu. 

52 5:05 PM 

A Single Baculovirus for the Production of rAAV8 Viral 

Vectors 

Lionel Galibert, Aurélien Jacob, Bérangère Langlet-Bertin, 

Marjorie Boutin Fontaine, Delphine Bonnin, Christophe Lecomte, 

Christel Rivière, Otto W. Merten. 

90 




Sensory (Ophthalmic and Auditory) Gene & 

Cell Therapy 

4:20 PM - 5:20 PM 

Room: 150 ABC 

53 4:20 PM 

Retinal Gene Therapy May Alter Connectivity of Visual 

Pathways 

M. Ashtari, L. Cyckowski, G. Zhang, P. Cook, K. Marshall, J. Wellman, 

J. Gee, A. Vossough, K. Shindler, A. Maguire, J. Bennett. 


54 4:35 PM 

Gene Therapy for Wet-AMD: Progress Report on a 

Phase I/II Clinical Trial 

Elizabeth P. Rakoczy, May C. Lai, Cora Pierce, Aaron L. Magno, 

Richard J. Samulski, David J. Dismuke, Joshua C. Grieger, Thomas 

W. Chalberg, Mark S. Blumenkrnz, Martyn French, Ian J. Constable. 

55 4:50 PM 

Preclinical Safety Studies for AAV2-MerTK Gene 

Therapy Vector for Retinitis Pigmentosa 

Thomas J. Conlon, Wen-Tao Deng, Renee C. Ryals, Kirsten E. Erger, 

Travis L. Cossette, Shannon E. Boye, Isaam McDoom, Nathalie 

Clemente, Brian Cleaver, Mark Potter, Corinne Abernathy, Sanford 

L. Boye, William H. Hauswirth. 

56 5:05 PM 

Glaucoma-GT, a Novel Gene Therapy Treatment for 

Primary Open-Angle Glaucoma 

Katie Binley, Scott Ellis, Vicky Scripps, Sharifah Iqball, Stuart 

Naylor, Kyriacos Mitrophanous. 


91


Abstract Directory, Thursday, May 16, 2013 


Clinical Gene & Cell Therapy Oral Abstract 

Session 

2:00 PM - 4:00 PM 


57 2:00 PM 

Acid alpha-Glucosidase Gene Replacement Therapy 

to the Diaphragm in Ventilator-Dependent Pompe 

Disease: One-Year Respiratory Motor Outcomes 

Barbara K. Smith, Daniel Martin, Lee Ann Lawson, Cathryn S. Mah, 

Saleem Islam, Thomas Conlon J. Conlon, Dawn Phillips, Shelley 

Collins, Barry J. Byrne. 

58 2:15 PM 

Long Term CD4 Reconstitution in HIV Subjects 

Receiving ZFN CCR5 Modiied CD4 T-Cells (SB-728-T) 

May Be Attributed to the Sustained Durability of the 

Central Memory T-Cell Subset 

Gary K. Lee, Joumana Zeidan, Jay Lalezari, Ronald Mitsuyasu, 

Shelley Wang, Marty Giedlin, Geoff Nichol, Winson Tang, Dale 

Ando, Raick-Pierre Sekaly. 

59 2:30 PM 

Cardiovascular Toxicity and Titin Cross-Reactivity of 

Afinity-Enhanced TCR-Engineered T Cells Against 

HLA-A1 Restricted MAGE-A3 Antigen 

Marcela V. Maus, Gerald P. Linette, Edward A. Stadtmauer, Aaron 

P. Rapoport, Bruce L. Levine, Lyndsey A. Emery, Leslie Litzky, 

Gwendolyn K. Binder-Scholl, Dominic P. Smethurst, Andrew B. 

Gerry, Nick J. Pumphrey, Alan Bennet, Joanna Brewer, Jane Harper, 

Namir Hassan, Bent K. Jakobsen, Michael Kalos, Carl H. June. 

60 2:45 PM 

Gene Therapy for Aromatic L-Amino Acid 

Decarboxylase Deiciency: Report of Treatment for 8 

Patients 

Wuh-Liang Hwu, Shin-ichi Muramatsu, Sheng-Hong Tseng, Kai- 

Yuan Tzen, Ni-Chung Lee, Yin-Hsiu Chien, Richard O. Snyder, Barry 

Byrne, Chun-Hwei Tai, Ruey-Meei Wu. 

61 3:00 PM 

Safe G-CSF Mobilization of Thalassemia Patient CD34+ 

Cells and Effective Globin Gene Transduction for 

Therapeutic Infusion 

Farid Boulad, Xiuyan Wang, Clare Taylor, Jinrong Qu, Leda Ferro, 

Garie Karponi, Shirley Bartido, Patricia Giardina, Glenn Heller, 

Susan E. Prockop, Aurelio Maggio, Michel Sadelain, Isabelle 

Riviere. 

62 3:15 PM 

Hematopoietic Stem Cell Gene Therapy with 

Lentiviral Vector in 4 Patients with Cerebral X-Linked 

Adrenoleukodystrophy: Long-Term Outcome and 

Comparison of Eficacy with Allogeneic Hematopoietic 

Stem Cell Transplantation 

Paul Orchard, Nathalie Cartier, Salima Hacein-Bey-Abina, 

Joanne Kurtzberg, Maria Escolar, Jakub Tolar, André Baruchel, 

Jean-Hughes Dalle, Gérard Michel, Stéphane Blanche, Cynthia 

Bartholomä, Manfred Schmidt, Pierre Bougnères, Christof van 

Kalle, Marina Cavazzana-Calvo, Alain Fischer, Patrick Aubourg. 

676 3:30 PM 

Update on Toca 511 - a retroviral replicating vector in 

clinical trials for High Grade Glioma 

Douglas J Jolly, Joan M. Robbins, Derek Ostertag, Amy Lin, Carlos 

Ibanez, Harry E. Gruber, Noriyuki Kasahara, Timothy F. Cloughesy, 

Manish K. Aghi, Susan M. Chang, Michael A. Vogelbaum, Santosh 

Kesari, Tom Mikkelsen, Joseph Landoli, E. Antonio Chiocca, James 

Elder, Greg Foltz, Dan Pertschuk 

299 3:45 PM 

Clinical Responses in Patients Infused with 

T Lymphocytes Redirected To Target -Light 

Immunoglobulin Chain 

Carlos A. Ramos, Barbara Savoldo, Enli Liu, Adrian P. Gee, Zhuyong 

Mei, Bambi Grilley, Cliona M. Rooney, Helen E. Heslop, Malcolm K. 

Brenner, Gianpietro Dotti. 



2:00 PM - 4:00 PM 


63 2:00 PM 

Comprehensive Study of Lentiviral “Integrome” in WAS 

Gene Therapy Patients 

Luca Biasco, Cristina Baricordi, Francesca Dionisio, Andrea 

Calabria, Samantha Scaramuzza, Danilo Pellin, Clelia Di Serio, 

Christof Von Kalle, Manfred Schmidt, Eugenio Montini, Luigi 

Naldini, Alessandro Aiuti. 

64 2:15 PM 

In Vivo Safety Evaluation of a Novel Insulated SIN LV 

Reveals Genotoxicity Escape-Mechanisms 

Monica Volpin, Daniela Cesana, Alexandre Artus, Caroline Duros, 

Fabrizio Benedicenti, Lucia Sergi Sergi, Luigi Naldini, Odile Cohen- 

Haguenauer, Eugenio Montini. 

65 2:30 PM 

No Impact of Lentiviral Transduction on Hematopoietic 

Stem and Progenitor Cell Telomere Length or Gene 

Expression Proile in the Rhesus Macaque 

Stephanie E. Sellers, Bogden Dumitriu, Mary Morgan, Colin O. Wu, 

Nalini Raghavarchari, Yanqin Yang, Naoya Uchida, John F. Tisdale, 

Robert E. Donahue, Andre Larochelle, Cynthia E. Dunbar. 

92 





2:00 PM - 4:00 PM 


66 2:45 PM 

Rescue of the X-CGD Phenotype by an Alpharetroviral 

Vector without Concomitant Induction of Aberrant 

Splicing 

Kerstin B. Kaufmann, Christian Brendel, Julia D. Suerth, Uta 

Müller-Kuller, Linping Chen-Wichmann, Joachim Schwäble, 

Shweta Pahujani, Hana Kunkel, Axel Schambach, Christopher 

Baum, Manuel Grez. 

67 3:00 PM 

Gene Transfer to Motor Neurons with Novel Targeted 

Lentiviral Vectors 

Ioanna Eleftheriadou, Antonio Trabalza, Stuart M. Ellison, Nicholas 

D. Mazarakis. 

68 3:15 PM 

NXF1 Controls Nuclear Export of Spliced and Unspliced 

Gammaretroviral Transcripts 

Toshie Sakuma, Yasuhiro Ikeda. 

69 3:30 PM 

Dual microRNA-Regulated Oncolytic Coxsackievirus B3 

Infection Displays Antitumor Activity with Attenuated 

Pathogenicity in Mice 

Shohei Miyamoto, Hiroyuki Inoue, Miyako Sagara, Beibei Wang, 

Koichi Takayama, Hiroyuki Shimizu, Yoichi Nakanishi, Kenzaburo 

Tani. 

70 3:45 PM 

Retroviral Replicating Vector (RRV)-Mediated Prodrug- 

Activator Gene Therapy Transcriptionally Targeted to 

Prostate Cancer 

Shuichi Kamijima, Takahiro Kimura, Kazunori Haga, Kei Hiraoka, 

Akihito Inagaki, Masamichi Takahashi, Christopher R. Logg, 

Chinghai Kao, Bernard H. Bochner, Noriyuki Kasahara. 


AAV Vector Biology 

2:00 PM - 4:00 PM 


71 2:00 PM 

The Structure of AAVrh32.33, a Novel Gene Delivery 

Vector 

Kyle Mikals, Hyun-Joo Nam, Kim Van Vliet, Luk H. Vandenberghe, 

Lauren E. Mays, Robert McKenna, James M. Wilson, Mavis 

Agbandje-McKenna. 

72 2:15 PM 

Identiication of Nucleolar Localization Sequences in 

the C-Terminal Region of Assembly-Activating Protein 

Lauriel F. Earley, Xiao-Xin Sun, Kei Adachi, Mushui Dai, Hiroyuki 

Nakai. 

73 2:30 PM 

Integrins Play a Pivotal Role in Transvascular 

Transport and Tissue Uptake of AAV9 In Vivo 

Shen Shen, Aravind Asokan. 

74 2:45 PM 

Adeno-Associated Virus Utilizes Host Cell Nuclear 

Import Machinery to Enter the Nucleus 

Sarah C. Nicolson, Richard J. Samulski. 

75 3:00 PM 

Functional Roles of the AAV2 Inverted Terminal Repeat 

in Messenger RNA Transport and Transgene Expression 

Yuan Wang, Yuan Lu, Lina Wang, Joon-Hyung Kim, George V. 

Aslanidi, Changquan Ling, Arun Srivastava, Chen Ling. 

76 3:15 PM 

Epigenetic Alterations in the Dystrophic Muscle 

Environment Down-Regulate rAAV-Mediated Transgene 

Expression 

Jean Baptiste Dupont, Benoit Tournaire, Christophe Georger, 

Laetitia Van Wittenberghe, Beatrice Marolleau, Laurence Dubreil, 

Emilie Lecomte, Fulvio Mavilio, Richard O. Snyder, Philippe 

Moullier, Adrien Leger. 

77 3:30 PM 

Sialic Acids and Erythrocyte Interactions Are Critical 

Determinants of Cardiopulmonary Tropism Displayed 

by AAV Serotype 4 

Shen Shen, Andrew Troupes, Nagesh Pulicherla, Aravind Asokan. 

78 3:45 PM 

Simultaneous Pharmacokinetic Proiling of Multiple 

AAV Serotypes and Mutants in a Non-Human Primate 

by AAV Barcode-Seq 

Kei Adachi, Yasuhiro Kawano, Gregory A. Dissen, Sergio R. Ojeda, 

Hiroyuki Nakai. 



2:00 PM - 4:00 PM 


79 Abstract Withdrawn from Presentation 

470 2:00 PM (Moved from Poster Session II) 

Human Parthenogenetic Stem Cell-Derived Hepatocytes 

for Liver Repopulation: Cell Therapy Demonstrated in a 

Pilot Study with Gunn Rats 

Larisa Agapova, Alina Ostrowska, Trudy Christiansen-Weber, 

Tiffany Chu, Ruslan Semechkin. 

80 2:15 PM 

Transplanted Mesenchymal Stem Cells Provide Type 

VII Collagen in the Skin of Recessive Dystrophic 

Epidermolysis Bullosa 

Katsuto Tamai, Shin Iinuma, Eriko Ikawa, Yasuhi Kikuchi, Ryo 

Fujita, Masaru Ishii, John McGrath, Jouni Uitto, Yasuhumi Kaneda. 



93




2:00 PM - 4:00 PM 




2:00 PM - 4:00 PM 



81 2:30 PM 

“Semi-Universal Donor Stem Cells” That Express a 

Single Class I Human Leukocyte Antigen (HLA) A*0201 

Can Present Peptides to Allorestricted CD8+ T-Cells 

German Gornalusse, Marvin Festag, Roli Hirata, Laura Riolobos, 

Cameron Turtle, Stan Riddell, David W. Russell. 

82 2:45 PM 

Targeted Correction of X-CGD iPSCs by TALEN-Mediated 

Gene Transfer to the CYBB Locus 

Colin L. Sweeney, Uimook Choi, Jizhong Zou, Suk See DeRavin, 

Harry L. Malech. 

83 3:00 PM 

Genetically Corrected Myoblasts Derived from 

Dystrophic hIPSCs Fused with Existing Muscle Fibers 

in Immunodeicient mdx Mice and Led to Expression of 

Micro-Dystrophin and a-Sarcoglycan 

Chantale Maltais, Carl Lebel, Joël Rousseau, Jacques P. Tremblay. 

84 3:15 PM 

Generation of Functional Monocyte/Macrophages 

by Genetic Correction of Patient-Speciic iPSC in 

Congenital Pulmonary Alveolar Proteinosis 

Nico Lachmann, Christine Happle, Doreen Lüttge, Mania 

Ackermann, Adele Mucci, Nicolaus Schwerk, Martin Wetzke, Sylvia 

Merkert, Axel Schambach, Gesine Hansen, Thomas Moritz. 

85 3:30 PM 

Clonogenic Hepatocyte Progenitors from the Liver, 

Pancreas and Gall Bladder 

Craig Dorrell, Meritxell Huch, Yuhan Wang, Hans Clevers, Markus 

Grompe. 

86 3:45 PM 

The Activation of the Wnt3a/b-Catenin Pathway 

Induced Maturation of Impaired Neutrophils as Well 

as the Endoplasmic Reticulum Stress in Induced 

Pluripotent Stem Cell Derived from a Severe Congenital 

Neutropenia Patient with ELANE Mutation 

Takafumi Hiramoto, Yasuhiro Ebihara, Yoko Mizoguchi, Kazuhiro 

Nakamura, Kiyoshi Yamaguchi, Kazuko Ueno, Naoki Nariai, Shinji 

Mochizuki, Shohei Yamamoto, Masao Nagasaki, Yoichi Furukawa, 

Kenzaburo Tani, Hiromitsu Nakauchi, Masao Kobayashi, Kohichiro 

Tsuji. 

87 2:00 PM 

The Effect of Neonatal Gene Therapy on Dysostosis 

Multiplex in Mucopolysaccaridosis VII Dogs after a 

Decade 

Katherine P. Ponder, Elizabeth M. Xing, Van W. Knox, Patricia A. 

O’Donnell, Yuli Liu, Susan Wu, Mark E. Haskins. 

88 2:15 PM 

scAAV Provides Sustained Expression of a Homologous 

Therapeutic Transgene in Large Mammalian Joints, 

with Enhanced Expression in Osteoarthritis 

Rachael S. Watson, Ted Broome, Andrew Smith, Patrick Colahan, 

Steven Ghivizzani. 

89 2:30 PM 

Dual Axis Gene-Therapy Using Stem Cells 

Overexpressing TGF-b3 in Combination with IL-1b and 

TNF-a RNA Silencing for Osteoarthritis Control in a 

Large Animal Osteochondral Chip Fracture Model 

Alan J. Nixon, Ashlee E. Watts, Laila Begum. 

90 2:45 PM 

Respiratory Function in a Canine Model of X-Linked 

Myotubular Myopathy Following Regional Limb 

Infusion with Recombinant AAV8-MTM1 

Melissa Goddard, Romain Joubert, Karine Poulard, Robert Grange, 

Michael Lawlor, Christelle Moal, Thibaud Jamet, Nathalie Danièle, 

Samia Martin, Christel Rivière, Caroline Hammer, Laetitia Van 

Wittenberghe, Erin Mitchell, Alban Vignaud, Carole Masurier, 

Philippe Moullier, Alan Beggs, Anna Buj-Bello, Barbara Smith, 

Barry Byrne, Martin Childers. 

91 3:00 PM 

Vascular Delivery of AAV.Micro-Dystrophin Using 

Whole Limb Re-Circulation in the Non-Human Primate 

as a Therapy for DMD 

Louis G. Chicoine, Chrystal L. Montgomery, Danielle A. Grifin, 

Kristin N. Heller, Kim Shontz, Thomas J. Preston, William G. 

Bremer, Christopher M. Walker, K. R. Clark, Jerry R. Mendell, 

Louise R. Rodino-Klapac. 

92 3:15 PM 

Safety and Histological Signs of Eficacy Following 

Systemic AAV9-CMV-Microutrophin Gene Therapy in a 

Canine Model for Duchenne Muscular Dystrophy 

Alock Malik, Andrew Mead, Mike Petrov, Marilyn Mitchell, Leonard 

Su, Frederick Balzer, Jacqueline Farag, Janet Bogan, Dan Bogan, Joe 

Kornegay, Hansell Stedman. 

93 3:30 PM 

A Xenograft Model of Skeletal Muscle for Preclinical 

Gene Therapy Studies 

Kathryn R. Wagner, Tracy Y. Zhang, Terence A. Partridge, Takako 

Jone, Oliver King, Fedik Rahimov, Qin Su, Guangpin Gao, Louis M. 

Kunkel, Robert H. Brown, Jr, Miguel Sena-Esteves. 

94 





2:00 PM - 4:00 PM 


94 3:45 PM 

Kidney Toxicity Caused by NEMO-Binding Domain 

(NBD) Peptide Therapy in a Murine Model of Muscular 

Dystrophy 

Paula R. Clemens, Daniel P. Reay, Katy Wack, Donna B. Stolz, Paul 

D. Robbins. 



2:00 PM - 3:00 PM 

Room: 150 ABC 

95 2:00 PM 

FunSel: Generation of AAV-Based, Arrayed Libraries for 

In Vivo Functional Selection of Secreted Factors and 

miRNAs Against Degenerative Disorders 

Giulia Ruozi, Francesca Bortolotti, Luca Braga, Antonella Falcione, 

Matteo Dal Ferro, Serena Zacchigna, Lorena Zentilin, Mauro 

Giacca. 

96 2:15 PM 

Intra-Infarct Delivery of Microparticles Containing 

MAP4K4 siRNA Reduce Acute Post-Infarct 

Inlammatory Responses 

Matthew S. Weaver, Jun Luo, James E. Dennis, Myriam Aouadi, 

Timothy P. Fitzgibbons, Shinya U. Amano, Michael P. Czech, 

Margaret D. Allen. 

97 2:30 PM 

Gene Transfer Using a Snake Venom Derived 

Vascular Endothelial Growth Factor Induces Robust 

Angiogenesis and Improves Ventricular Function in 

Ischemic Porcine Myocardium 

Paavo J. Halonen, Jussi Nurro, Antti Kuivanen, Tuomas T. Rissanen, 

Seppo Ylä-Herttuala. 

98 2:45 PM 

Long-Term Robust Myocardial Transduction of the Dog 

Heart from Peripheral Vein by Adeno-Associated Virus 

Serotype-8 (AAV-8) 

Xiufang Pan, Yongping Yue, Keqing Zhang, William Lostal, Jin-Hong 

Shin, Dongsheng Duan. 



2:00 PM - 3:15 PM 


99 2:00 PM 

Effective Gene Electrotransfer Is Dependent on the 

Pulse Parameters of the Applied Electric Field 

Richard Heller, Cathryn Lundberg, Fanying Li, Niculina Burcus, 

Shawna Shirley. 

100 2:15 PM 

Targeted Modulation of BACE1 Expression and 

Electrical Activity in Neuronal Cultures Using Voltage 

Pre-Conditioning 

Arati M. Sridharan, Jit Muthuswamy. 

101 2:30 PM 

Ultrasound and Microbubbles Mediated Factor VIII 

Plasmid Gene Therapy in Hemophilia A Mice 

Shuxian Song, Misty L. Noble, Ryan Sun, Luping Fan, Carol H. Miao. 

102 2:45 PM 

Gadolinium Chloride Extends the Duration of Gene 

Expression in Dogs Hydrodynamically Infused with 

Sleeping Beauty Transposons 

R. Scott McIvor, Myra C. Rusten, Erik R. Olson, Jason B. Bell, Elena 

L. Aronovich, Kendra A. Hyland, John R. Lake, Roland Gunther, 

Bryan M. Ladd, David W. Hunter, Perry B. Hackett. 

103 3:00 PM 

Non-Invasive In Vivo Imaging, Biodistribution and 

Clearance Analyses of Intravenously Administered 

Extracellular Vesicles 

Charles P. Lai, Osama Mardini, Casey A. Maguire, Bakhos T. 

Tannous, Xandra O. Breakeield. 



3:05 PM - 4:05 PM 

Room: 150 ABC 

104 3:05 PM 

Maternal Immunization with Chimpanzee Adenovirus 

Expressing RSV Fusion Protein Protects Against 

Neonatal RSV Pulmonary Infection 

Anurag Sharma, Rebecca Wendland, Biin Sung, Wendy Wu, Stefan 

Worgall. 



95




3:05 PM - 4:05 PM 

Room: 150 ABC 

Poster Session I 

4:00 PM - 6:00 PM 

Room: Exhibit Hall C/D 

AAV Vectors I 



432 3:20 PM Moved from Poster Session II 

A Novel Cancer Cell Vaccine Using Induced Pluripotent 

Stem Cells Genetically Engineered To Produce GM-CSF 

Elicits Substantial Antitumor Immunity in a Syngeneic 

Mouse Model 

Hiroyuki Inoue, Ayumi Wanatabe, Chika Sakamoto, Megumi 

Narusawa, Shohei Miyamoto, Makoto Inoue, Koichi Takayama, 

Mamoru Hasegawa, Yoichi Nakanishi, Kenzaburo Tani. 

106 3:35 PM 

Safety, Toxicology and Eficacy of a Lung-Directed AAV- 

Based Gene Therapy for a1-Antitrypsin Deiciency 

Maria J. Chiuchiolo, Neil R. Hackett, Esther Z. Frenk, Yihharn 

Hwang, Jianping Qiu, Janet Benson, Gensheng Wang, Stephen M. 

Kaminsky, Ronald G. Crystal. 

107 3:50 PM 

Allele-Preferred Targeted Correction of CFTR Gene in 

Cystic Fibrosis Induced Pluripotent Stem Cells 

Jacquelin H. Bui, Ana M. Crane, Xuan S. Li, Philipp Kramer, Manuel 

Gonzalez-Garay, Wei Liao, Jianbin Wang, Helena C. Sun, David E. 

Paschon, Dmitry Y. Guschin, Philip D. Gregory, Darrell N. Kotton, 

Michael C. Holmes, Brian R. Davis. 


Immunologic & Host Responses in Gene & Cell 


3:20 PM - 4:05 PM 


108 3:20 PM 

Escaping Immune Activation through the Use of CpG- 

Depleted AAV Vectors 

Susan Faust, Peter Bell, Benjamin Cutler, Scott Ashley, Joseph 

Rabinowitz, James Wilson. 

109 3:35 PM 

Ex Vivo Expanded Treg Suppress Inhibitor Formation in 

Protein and Gene Therapies for Hemophilia 

Debalina Sarkar, David M. Markusic, Todd M. Brusko, Roland W. 

Herzog. 

110 3:50 PM 

Overcoming Pre-Existing Humoral Immunity to AAV 

Using Capsid Decoys 

Federico Mingozzi, Xavier M. Anguela, Giulia Pavani, Yifeng Chen, 

Robert H. Davidson, Daniel H. Hui, Mustafa Yazicioglu, Liron 

Elkouby, Christian J. Hinderer, Armida Faella, Carolann Howard, 

Alex Tai, Gregory M. Podsakoff, Margaret C. Ragni, Shangzhen 

Zhou, Etiena Basner-Tschakarjan, Fraser J. Wright, Katherine A. 

High. 

111 

Locoregional Intravenous Delivery of AAV Vector in 

Macaque Results in Persistent Transgene Expression 

Despite a Dedicated Humoral and Cellular Immune 

Responses 

Gwladys Gernoux, Marie Devaux, Laurence Dubreil, Nicolas Jaulin, 

Mickaël Guilbaud, Jack-Yves Deschamps, Thibaut Larcher, Guigand 

Lydie, Maeva Dutilleul, Caroline Le Guiner, Yan Chérel, Philippe 

Moullier, Oumeya Adjali. 

112 

Global CNS Gene Delivery in Non-Human Primates 

by Intra-CSF Administration: Evasion of Anti-AAV 

Neutralizing Antibodies but Not a Transgene-Speciic 

CTL Response 

Steven J. Gray, Sahana Nagabhushan Kalburgi, Thomas J. McCown, 

R. Jude Samulski. 

113 

Increased Occurrence of Hepatocellular Carcinoma 

(HCC) in Mice after Neonatal Adeno-Associated Virus 

(AAV8) Gene Delivery Exhibits Dose-Dependent 

Tumorigenesis 

Randy J. Chandler, Charles P. Venditti. 

114 

A Novel Adeno-Associated Virus Serotype (AAV)-8 

Capsid Mutant Improves Human Coagulation Factor IX 

Expression In Vivo 

Dwaipayan Sen, Rupali A. Gadkari, Govindarajan Sudha, Nishanth 

Gabriel, V. Ramya, Sukesh C. Nair, N. Srinivasan, Alok Srivastava, 

Giridhara R Jayandharan. 

115 

New MiniPromoters with Restricted-Retinal Expression 

When Docked in the Mouse Genome Show the Same 

Expression When Delivered in AAV 

Elizabeth M. Simpson, Charles N. de Leeuw, Frank M. Dyka, 

Sanford L. Boye, Stephanie Laprise, Michelle Zhou, Alice Y. Chou, 

Lisa Borretta, Simone C. McInerny, Elodie Portales-Casamar, 

Magdalena I. Swanson, Steven J. M. Jones, Robert A. Holt, Daniel 

Goldowitz, William W. Hauswirth, Wyeth W. Wasserman. 

116 

AAV Serotypes 1, 8, and 9 Share a Conserved 

Mechanism for Axonal Transport but Exhibit 

Differences in Distal Gene Transfer In Vivo 

Michael J. Castle, April Giles, Erika L. F. Holzbaur, John H. Wolfe. 

117 

AAV6 Captron Vectors Facilitate Persistent Expression 

of Therapeutic Levels of Human a1-Antitrypsin in 

Canine Lungs 

Christine L. Halbert, David K. Madtes, A. D. Miller. 

96 



Poster Session I, continued 

4:00 PM - 6:00 PM 


AAV Vectors I 

118 

Productive Life Cycle of the AAV2 in the Complete 

Absence of a Conventional Polyadenylation Signal 

Lina Wang, Yuan Wang, Yuan Lu, George V. Aslanidi, Arun 

Srivastava, Changquan Ling, Chen Ling. 

119 

A Unique Biological Property of an AAV9 Mutant 

Showing Increased Cell Attachment in the Presence and 

Absence of AAV5 

Kei Adachi, Alex Summers, Hiroyuki Nakai. 

120 

Effective Transduction of Common Marmoset with rAAV 

1 and 9 To Generate NHP Model of Muscular Dystrophy 

Hironori Okada, Hidetoshi Ishibashi, Hiromi Hayashita-Kinoh, 

Tomoko Chiyo, Yuko Nitahara-Kasahara, Takashi Okada, Shin’ichi 

Takeda. 

121 

Precise Evaluation of NAb Status Against Adeno- 

Associated Viral Vectors and an Approach toward 

Managing Its Inhibitory Effect 

Hiroaki Mizukami, Jun Mimuro, Shuji Hishikawa, Tomokazu 

Ikemoto, Ryosuke Uchibori, Tomonori Tsukahara, Masashi Urabe, 

Akihiro Kume, Asuka Sakata, Tsukasa Ohmori, Seiji Madoiwa, 

Yoichi Sakata, Keiya Ozawa. 

122 

Dissection of Key Parameters Inluencing Ex Vivo AAV 

Library Selections 

Eike Kienle, Dirk Grimm. 

123 

Peptide-Mediated Retargeting of 12 AAV Serotypes 

Anna Sacher, Kathleen Börner, Eike Kienle, Hans-Georg 

Kraeusslich, Martin Mueller, Dirk Grimm. 

124 

Creation of Liver-Detargeting AAV2-Derived Mutants 

Based on the Knowledge of AAV9 Capsid Functions 

Kei Adachi, Michael Verga, Hiroyuki Nakai. 

125 

Cruciform Hairpin Structure of AAV Genome Is 

Speciically Subject to ATM-Dependent Silencing of 

Gene Expression 

Marcela P. Cataldi, Kim Zaraspe, Haiyan Fu, Douglas M. McCarty. 


126 

Shielding the Hexon Hyper Variable Region 1 of 

Ad5-Based Gene Transfer Vectors by PEGylation 

Decreases Sequestration by Erythrocytes and Increases 

Blood Coagulation Factor XMediated Hepatocyte 

Transduction 

Lea Krutzke, Jan-Michael Prill, Tatjana Engler, Stefan Kochanek, 

Florian Kreppel. 

127 

Therapeutic Eficacy in a Breast Cancer Model of Novel 

Conditionally Replicative Adenovirus (CRAd) Based 

upon the Deletion of Minor Core Protein Gene V 

Hideyo Ugai, George C. Dobbins, Minghui Wang, David T. Curiel. 

128 

No Recombinant Vector Sequences Detected in 

Tumor Tissues from Two Research Subjects Received 

Adenovirus Ornithine Transcarbamylase Gene Therapy 

Li Zhong, Shaoyong Li, Mengxin Li, Yu Zhang, Brendan Lee, Mark L. 

Batshaw, James M. Wilson, Guangping Gao. 

129 

Oncolytic Adenovirus Expressing IL-23 and p35 

Elicits IFN-g- and TNF-a-Co-Producing T Cell-Mediated 

Antitumor Immunity 

Il-Kyu Choi, Yan Li, Eonju Oh, Chae-Ok Yun. 

130 

Wnt Decoy Receptor (sLRP6E1E2) Induces Antiibrotic 

Effect Via Inhibition of Wnt Signaling in Keloid Scars 

Jung-Sun Lee, Won Jai Lee, Chae-Ok Yun. 

131 

Versican Stimulates Adenoviral Transgene Expression 

in a JAK Dependent Manner 

Patricia Y. Akinfenwa, Wesley S. Bond, Mary Y. Hurwitz, Richard L. 

Hurwitz. 

132 

Replication of Human Adenoviruses in Non-Human 

Cells In Vitro and In Vivo 

Catherine M. Crosby, Mallory A. Turner, Michael A. Barry. 

133 

Adenovirus Serotypes Differ in Their Susceptibility to 

Neutralization by Mouse Serum: Role of Coagulation 

Factor X 

Jie Tian, Zhili Xu, Andrew P. Byrnes. 

134 

Non-Covalent Coating of Adenoviral Vectors with 

PAMAM Dendrimer Conjugates Allows for CAR 

Independent Virus Uptake and Targeting to the EGF 

Receptor 

Alexandra Vetter, Kulpreet S. Virdi, Sigrid Espenlaub, Wolfgang 

Rödl, Ernst Wagner, Florian Kreppel, Christine Spitzweg, Manfred 

Ogris. 



97




4:00 PM - 6:00 PM 



135 

A Cell Line for Large-Scale Production of 

Nondisseminative Adenoviral Vectors (Deleted 

of Protease [PS] Gene) for Cancer Therapy and 

Vaccination 

Rénald Gilbert, Claire Guilbault, Mélanie Simoneau, Seyyed Mehdy 

Elahi, Bernard Massie. 

136 

Inhibition of Breast Cancer Malignant Progression by 

Neutralizing Hypoxia-Induced Cell Motility 

Yi Lu, Liyuan Li, Jun Zhang. 

137 

HATHFUN: A Protein-Based Alternative to Viral 

Reprogramming 

Trudy Christiansen-Weber, Rodolfo Gonzalez, Alexa Dillberger, 

Tiffany Chu, Larisa Agapova, Alina Ostrowska, Ruslan Semechkin. 

138 

hChonJb#7 Cells (Chondrocytes Expressing TGF-b1) 

Reduced Pain in a Rat Osteoarthritis Model 

Kyoung Baek Choi, Hyeon Youl Lee, Dae Wook Kim, Su Jeong Kim. 

561 (Moved from Poster Session III) 

Lister Strain Vaccinia Virus Demonstrates Favourable 

Anti-Tumor Eficacy and Tumor-Selectivity Compared 

to Western Reserve Strain 

Jonathan Hughes, Pengju Wang, Ghassan Alusi, Vipul Bhakta, 

Yongchao Chu, Hailiang Shi, Iain McNeish, Andrea McCart, Istvan 

Fodor, Nicholas Lemoine, Yaohe Wang. 


139 

Electro-Chemo-Gene Therapy for Long-Term 

Management of Cancer in Canines 

Jeffry Cutrera, Xueqing Xia, Glenn King, Pamela Jones, Kristin 

Kicenuik, Shulin Li. 

140 

Engineering Novel Transducers for Effective 

Ultrasound-Mediated Gene Therapy 

Misty L. Noble, Shuxian Song, Ryan R. Sun, Kyle P. Morrison, 

George W. Keilman, Luping Fan, Marla Paun, Keith R. Loeb, Carol 

H. Miao. 

141 

Gene Transfer Mediated by First-Generation Plasmids 

Induce Large Scale Proteomic Changes to Cellular 

Programming That Are Minimized by Elimination of 

Prokaryotic Sequences 

Ramaz Geguchadze, Zhimin Wang, Lee Zourelias, Laurie Machen, 

Michael J. Passineau. 

142 

Development of pDNA-Loaded Bubble Liposomes for 

a Potential Theranostic Approach: Nano-Sized Agents 

Containing Cationic Lipid for Ultrasound Imaging and 

Gene Delivery 

Yoichi Negishi, Yoko Endo-Takahashi, Daichi Suzuki, Ukai Saori, 

Norio Takagi, Ryo Suzuki, Kazuo Maruyama, Yukihiko Aramaki. 

143 

Intracarotid Injection of Adipose Tissue-Derived 

Platelet-Derived Growth Factor Receptor b-Positive 

Cells in Rat and Canine Model: Feasibility of Cell 

Delivery under Mannitol-Induced Blood-Brain Barrier 

Opening 

Jung-Ick Byun, Keun-Hwa Jung, Kon Chu, Sung Won Yoon, Jong 

Young Lee, Soon-Tae Lee, Jeong-Min Kim, Manho Kim, Sang Kun 

Lee, Moon-Hee Han, Jae-Kyu Roh. 

144 

Measuring Intracellular Degradation of Polylactide- 

Based Magnetic Nanoparticles Using FRET 

Jillian E. Tengood, Alferiev S. Ivan, Fishbein Ilia, Robert J. Levy, 

Michael Chorny. 

145 

Gene Delivery System for Liver by Sonoporation with 

Nanobubble 

Ryo Suzuki, Yusuke Oda, Daiki Omata, Yoshikazu Sawaguchi, Yoichi 

Negishi, Kazuo Maruyama. 

146 

Exploring Therapeutic Applications of Pseudouridine- 

Containing mRNA 

Katalin Karikó, Hiromi Muramatsu, Norbert Pardi, Drew 

Weissman. 

147 

Transfection of Pseudouridine-Containing mRNA 

Encoding CPD-Photolyase into UVB-Irradiated Human 

Keratinocytes Results in Rapid Repair of DNA Damage 

Gábor Boros, Edit Miko, Eszter Emri, Gijsbertus T. J. van der Horst, 

Iren Horkay, Gabriella Emri, Eva Remenyik, Hiromi Muramatsu, 

Drew Weissman, Katalin Karikó. 

148 

Improvement of Both Cell Survival and Eficacy for 

Large Plasmids Electrotransfer in MSC 

Léa L. Lesueur, Lluis M. Mir, Franck M. Andre. 

149 

Minicircles Mediate Superior Ultrasound-Assisted 

Gene Transfer Relative to First-Generation Plasmids at 

Eqimolar Concentrations 

Zhimin Wang, Ramaz Geguchadze, Lee Zourelias, Laurie Machen, 

Michael J. Passineau. 

98 




4:00 PM - 6:00 PM 



150 

Multifunctional Cationic Lipid-Based Magnetic 

Nanoparticles for Enhanced Gene Transfection and 

Magnetic Resonance Imaging Applications 

Srinath Govindarajan, Kumiko Kitaura, Makoto Takafuji, Hirotaka 

Ihara, K. S. Varadarajan, Anant B. Patel, Vijaya Gopal. 

158 

Anionic, Self-Assembling Nanocomplexes for Improved 

Receptor-Targeted Transfection In Vitro and In Vivo 

Aristides D. Tagalakis, Gavin D. Kenny, Alison S. Bienemann, John 

A. T. Pugh, Mark F. Lythgoe, Cameron W. McLeod, Edward A. White, 

Stephen L. Hart. 

159 

Prostate Cancer Cell-Speciic siRNA and Drug Co- 

Delivery with Aptamer-Functionalized Liposomes 

(Aptamosomes) 

Gwang-Hyun Lee, Hwa Yeon Jeong, Yong Serk Park, Dong-Eun Kim, 

Keun-Sik Kim. 

151 

Non-Viral Enteral Hormone Gastrin Gene Therapy 

Improves the Function and Stimulates the Proliferation 

of Pancreatic beta Cells 

Jeonghyun Park, Hyesook Jung, Na Han, Taekyun Kim, Minjung 

Kwon, Soonhee Lee, Byungdoo Rhee, Mikyung Kim. 

152 

Initial Toxicology of High Dose GNE Plasmid in Rodent 

and Canine Animal Species 

Yadira Valles-Ayoub, Rosangela Carbajo, Lucia Sandoval, Zeshan 

Khokher, Jorge Garcia-Figueroa, Sarah Stein, Daniel No, Babak 

Darvish, Daniel Darvish. 

153 

Elucidating the Expression Kinetics and Iniltration 

Resulting from Gene Delivery Enhanced by Dermal 

Electroporation 

Janess M. Mendoza, Gleb Kichaev, Dinah Amante, Christine Knott, 

Trevor R. F. Smith, Niranjan Y. Sardesai, Kate E. Broderick. 

154 

The Optimization of Electroporation Parameters To 

Enhance the Eficiency of Plasmid Gene Delivery in 

Muscle 

Ningqing Wang, Yong Diao. 

160 

Large Gene Non-Viral Transfer Mediated by Chitosan 

Nanoparticles and Integrase 

Daniel C. Chung, Anna V. Oliviera, Hadassah Janumala, Gabriela 

Silva. 

161 

Co-Delivery of siRNA and pDNA for Increased 

Transfection Eficiency 

Laura Rose, Michael Hogarth, Hasan Uludag. 

162 

Stability of Polymer/Plasmid DNA Complexes In Vitro 

and In Vivo 

Laura Rose, Jeremy Fife, Cezary Kucharski, Hasan Uludag. 

163 

When Nucleic Acids Meet Cationic Polymers: Effects of 

Mixing Sequence on Complexation and Transfection 

Eficiencies 

Soo Kyung Cho, Xi Wang, Regina Ragan, Young Jik Kwon. 

164 

Bioreducible and Acid-Labile Poly(Amido Amine)s for 

Eficient Gene Delivery 

Qinghui Zhou, Yezhi You, Zhiqiang Yu, Junjie Yan.w 



155 

A High Throughput Screening Approach To Identify 

Gene Transfer Peptides In Vitro 

Jing Li, Jason Duskey, Kevin G. Rice. 

156 

Targeted Delivery of Short Interfering RNA by Human 

TAR RNA-Binding Protein-Afibody Chimera 

Gulam H. Dar, Erica Nathan, Nalam M. Rao, Vijaya Gopal. 

157 

Oligopeptide Comb Architectures for Controlled DNA 

Binding and Non-Viral Transfection 

Sangram S. Parelkar, Rachel Letteri, Delphine Chan-Seng, Todd 

Emrick. 



166 

Micro-Dystrophin Ameliorates Muscular Dystrophy in 

the Canine Model of Duchenne Muscular Dystrophy 

Jin-Hong Shin, Xiufang Pan, Chady H. Hakim, Hsiao T. Yang, 

Yougping Yue, Keqing Zhang, Ronald L. Terjung, Dongsheng Duan. 

167 

Expression of Minidystrophin from the Dystrophin 

Locus by TALEN Mediated Gene Targeting 

David G. Ousterout, Pablo Perez-Pinera, Pratiksha I. Thakore, Xiao 

Xiao, Charles A. Gersbach. 


99




4:00 PM - 6:00 PM 



168 

Activation, Expansion, and In Vivo Regenerative 

Potential of Pax3-Transduced Satellite Cells: A Potential 

Cell-Based Therapy for Duchenne Muscular Dystrophy 

Antonio Filareto, Robert W. Arpke, Darko Bosnakovski, Radbod 

Darabi, Fabrizio Rinaldi, Auston Miller, Michael Kyba, Rita C. R. 

Perlingeiro. 

169 

Genetic Correction of Facioscapulohumeral Dystrophy 

Using Telomere Insertion: Potential for Therapeutic 

Application 

Fabrizio Rinaldi, Linn Hartweck, Darko Bosnakovski, Erik 

Toso, Abhijit Dandapat, Radbod Darabi, Thomas Zwaka, Simon 

Nargarkhan, Rita Perlingeiro, Michael Kyba. 

170 

Enhanced Myogenic Differentiation Induced by 

Engineered MyoD Transcription Factors 

Ami M. Kabadi, Pratiksha I. Thakore, Charles A. Gersbach. 

171 

Viral Mediated Acute Mouse Model for Myotonic 

Dystrophy Type1 

Syed-Rehan A. Hussain, Lindsay Wallace, Danielle Grifin, Chrystal 

Montgomery, Scott Q. Harper, Zarife Sahenk, K. Reed Clark. 

172 

Pre-Clinical Eficacy Study of Beta-Sarcoglycan Gene 

Transfer 

Eric R. Pozsgai, William E. Grose, Danielle A. Grifin, Kristin N. 

Heller, Jerry R. Mendell, Louise R. Rodino-Klapac. 

173 

The Cardiac Alkali Myosin Light Chain Can Restore 

Skeletal Muscle Function in a Mouse Model of Nemaline 

Myopathy 

Johan M. Lindqvist, Julien Ochala. 

176 

Engraftment of Mesenchymal Stromal Cells That Can 

Differentiate To Form Myogenic Cells Is Enhanced 

by Expressing IL-10 in Dog with Duchenne Muscular 

Dystrophy 

Yuko N. Kasahara, Hiromi H. Kinoh, Tomoko Chiyo, Hironori 

Okada, Takashi Okada, Shin’ichi Takeda. 

177 

Modulating Cell Cycle Inhibitor Expression To Enhance 

Skeletal Myoblast Engraftment 

Pratiksha I. Thakore, David G. Ousterout, Charles A. Gersbach. 

178 

Human Muscle Derived Stem Cells Exhibit Bone 

Regeneration Capacity Similar to Bone Marrow 

Mesenchymal Stem Cells When Genetically Modiied To 

Express BMP2 Both In Vitro and In Vivo 

Xueqin Gao, Arvydas Usas, Ying Tang, Aiping Lu, Jian Tan, Johannes 

Schneppendahl, Nick Oyster, Bing Wang, Rocky Tuan, Johnny 

Huard. 

179 

Rapid Progression of Muscular Dystrophy in 

Dystrophin/Utrophin-/- Mice Is Associated with Muscle 

Progenitor Cell Exhaustion 

Aiping Lu, Minakshi Poddar, Ying Tang, Jonathan D. Proto, 

Xiaodong Mu, Bing Wang, Johnny Huard. 

180 

A New Mouse Model of DMD: A Tool for Therapeutic 

Development Directed at Exon Duplications 

Adeline Vulin, Nicolas Wein, Yuuki J. Kaminoh, Andrew R. Findlay, 

Kevin M. Flanigan. 

181 

Investigation of Dystromirs in the mdx Mouse 

Thomas C. Roberts. 

182 

Dose-Dependent Toxicity of Humanized Renilla 

Reniformis GFP (hrGFP) Limits Its Utility as a Reporter 

Gene in Mouse Muscle 

Lindsay M. Wallace, Andrew Moreo, K. Reed Clark, Scott Q. Harper. 

174 

Generating Dystrophin+ Myogenic Cells from mdx 

Fibroblasts by Using a Triple-Recombinase iPS Cell- 

Based Strategy 

Jonathan M. Geisinger, Chunli Zhao, Alfonso P. Farrugio, 

Christopher L. Chavez, Christopher R. Bjornson, Tawny L. Neal, 

Michele P. Calos. 

175 

Alpha7 Integrin Partially Preserves the Mechanical 

Function of the Extensor Digitorum Longus Muscle in 

Dystrophin-Deicient Mice 

Chady H. Hakim, Dean J. Burkin, Dongsheng Duan. 


183 

Electroporation of ZFN mRNA Enables Eficient CCR5 

Gene Disruption in Mobilized Blood Hematopoietic 

Stem Cells at Clinical Scale 

Paula M. Cannon, Jill E. Henley, Travis Wood, Lynn Truong, Jianbin 

Wang, Kenneth Kim, Jenny Yan, Philip D. Gregory, Gary Lee, 

Michael C. Holmes. 

100 




4:00 PM - 6:00 PM 



184 

Next Generation TALENs Mediate Eficient Disruption of 

the CCR5 Gene in Human HSCs 

Nick Llewellyn, Jeffrey C. Miller, John Campo, Colin Exline, Orla 

Mulhern, Jill Henley, Jianbin Wang, Dmitry Guschin, Danny Xia, 

Irina Ankoudinova, Philip D. Gregory, Lei Zhang, Edward J. Rebar, 

Michael C. Holmes, Paula M. Cannon. 

185 

Enhanced Neutralising Antibody Response to Bovine 

Viral Diarrhoea Virus (BVDV) Induced by BVDV DNA 

Vaccine Co-Expressing RIG-I Agonist in Cattle 

Laila M. El-Attar, Olivia Anstaett, Carole Thomas, Jeremy Luke, 

James Williams, Joe Brownlie. 

186 

Preclinical Safety Evaluation of an Antibiotic-Free 

LAMP-1 Plasmid Allergy Immunotherapy 

Teri L. Heiland, James A. Williams, William G. Hearl. 

187 

Inhibition of HBV Replication through ZFN-Mediated 

Disruption of cccDNA 

Daniel Stone, Nick Weber, Harshana De Silva Feelixge, Keith R. 

Jerome. 

188 

Development of Designer Homing Endonucleases for 

Inactivation of the HIV Provirus 

Esther Mintzer, Jordan Jarjour, Andy Scharenberg, Barry Stoddard, 

David Baker, Nick Weber, Daniel Stone, Keith R. Jerome. 

189 

Whole Cell-SELEX Derived Anti-CD7 Aptamers for the 

Delivery of Anti-CCR5 siRNA: A Prohylactic Approach to 

HIV Therapy 

Laura Millroy, Daniel Lazar, Makobetsa Khati, Marc S. Weinberg. 

190 

Capsid Modiied AAV2 Vectors Are Capable of 

Generating Functional Cytotoxic T Cells in a Prime- 

Boost Vaccine Protocol 

Brad E. Hoffman, Irene Zolotukhin, Roland W. Herzog, Ou Cao. 

191 

The Anti-Genomic (Negative) Strand of Hepatitis C 

Virus Is Not Targetable by shRNA 

Leszek Lisowski, Menashe Elazar, Kirk Chu, Jeffrey S. Glenn, Mark 

A. Kay. 

192 

Development of the Oral Universal Type A Inluenza 

Vaccine Using Biidobacterium Displaying Inluenza- 

M2e Protein 

Naoya Morishita, Suzuka Ozaki, Koichi Kitagawa, Chihiro 

Shirosaki, Hiroki Saito, Katsumi Shigemura, Masato Fujisawa, 

Masato Kawabata, Toshiro Shirakawa. 

193 

Enzyme Engineering of Homing Endonucleases To 

Target HBV cccDNA 

Nicholas D. Weber, Ryo Takeuchi, Jordan Jarjour, Daniel Stone, 

Barry L. Stoddard, Keith R. Jerome. 

194 

In Vivo Electroporation of Minicircle DNA as a Noval 

Method of Vaccine Delivery 

Wenlin Huang, Qingtao Wang, Changchuan Pan, Shijuan Gao, Wei 

Jiang. 

195 

The Potential Therapeutic Role of Secreted Antiviral 

Entry Inhibitory (SAVE) Peptides Expressed by 

Transduced MSCs To Block Viral Replication 

Narae Lee, Edith Walker, Wenpeng Li, Lisa Egerer, Dorothee von 

Laer, Bruce A. Bunnell, Stephen E. Braun. 


196 Abstract Withdrawn 

197 

Dual Transgenesis of T Cells with a CD44v6-Speciic 

CAR and a Suicide Gene for the Safe Eradication of 

Myeloid Leukemia and Myeloma 

Monica Casucci, Benedetta Nicolis di Robilant, Laura Falcone, 

Barbara Camisa, Pietro Genovese, Bernhard Gentner, Claudio 

Bordignon, Barbara Savoldo, Fabio Cicieri, Luigi Naldini, 

Gianpietro Dotti, Chiara Bonini, Attilio Bondanza. 

198 

Capitalizing on Epitope Escape To Avoid T Cell 

Fratricide without Compromising Targeting of CD56+ 

Tumors 

Denise L. Crossland, Sonny Ang, Simon Olivares, Kirsten Switzer, 

Tiejuan Mi, Helen Huls, Laurence J. N. Cooper. 

199 

Phase II Study of FANG (Autologous Tumor Cell 

– GMCSF/pbi-shRNA Furin DNA Plasmid) Vaccine in 

Advanced Melanoma 

John Nemunaitis, Neil Senzer, Minal Barve, Padmasini Kumar, 

Donald D. Rao, Gladice Wallraven, Beena O. Pappen, Jospeh Kuhn, 

Peter Beitsch, Robert Steckler, Brian M. Gogel, Walton Taylor, 

Phillip B. Maples. 

200 

Renal Cell Tumor-Mediated Reprogramming of Natural 

Killer Cells by Soluble Immune Modulators 

Yue Guan, Purba Singh, Trenae Mann, Christopher B. Chambers, 

Donald S. Torry, Andrew Wilber. 



101




4:00 PM - 6:00 PM 



201 

A New Epithelial Junction Opener for Cancer Therapy 

Ines Beyer, Hua Cao, Jonas Persson, Roma Yumul, Andre Lieber. 

202 

A Chemotherapy-Selective Method for Expansion of 

Transgenic T Cells 

David Rushworth, Laurence Cooper. 

203 

Phase I Study of the “Triad” Autologous (FANG) 

Vaccine, Incorporating Bifunctional shRNAfurin and 

GMCSF Transgene Expression, in Advanced Ewing’s 

Sarcoma: Preliminary Data in Pediatrics Patients 

Maurizio Ghisoli, Carl Lenarsky, Gladice Wallraven, Padmasini 

Kumar, Phillip B. Maples, John Nemunaitis. 

204 

Dissecting the Human T Cell Receptor Repertoire 

in Different Immunological Conditions by Deep 

Sequencing 

Eliana Ruggiero, Jan P. Nicolay, Anne Arens, Raffaele Fronza, Anna 

Paruzynski, Ali Nowrouzi, Gökçe Ürenden, Sergij Goerdt, Hanno 

Glimm, Peter H. Krammer, Manfred Schmidt, Christof von Kalle. 

205 

Aptamer Targeted Antigen Delivery 

Matthew Levy, Brian C. Wengerter, Debborah Palliser, Joseph 

Katakowski. 

206 

FANG Cytokine Expression Analyses Update 

Padmasini Kumar, Connor Phalon, Beena O. Pappen, Gladice 

Wallraven, Yang Yu, Fabienne Norvell, Christopher M. Jay, Zhaohui 

Wang, Donald D. Rao, John Nemunaitis, Neil Senzer, Phillip B. 

Maples. 

207 

Effect of Interleukin-15 and Dendritic Cells on the 

Phenotype and Cytotoxicity of Cytokine-Induced Killer 


Xianghui He, Zhaohui Lai, Na Zhao, Liwei Zhu. 

208 

Investigating Macrophage Induction of Metastasis: A 

Precursor To Identify Therapeutic Targets 

Trevor Memmott, Evita Weagel, Justin Livingston, Pingguo Liu, Atif 

Elnaggar, Richard Robison, Kim O’Neill. 


Cancer-Targeted Gene & Cell Therapy: Viral 

Vector / siRNA-Mediated Therapies 

210 

Modelling Viral Insertion Sites Via Network Based 

Frameworks 

Raffaele Fronza, Cynthia Bartholomae, Annette Deichmann, 

Stephanie Laufs, Christof von Kalle, Manfred Schmidt. 

211 

rAAV Mediated Modulation of miR-1 Inluences the 

Progression of Hepatocellular Carcinoma in a Mouse 

Model of Alpha-1 Antitrypsin 

Florie Borel, Qiushi Tang, Nancy Marcus, Jeffery Teckman, Terence 

Flotte, Christian Mueller. 

212 

AAV Mediated IFNb Gene Therapy of Orthotopic 

Xenograft Mouse Model of Invasive Glioblastoma 

multiforme 

Dwijit GuhaSarkar, James Neiswender, Miguel Sena-Esteves. 

213 

Response of Drug Sensitive and Resistant Breast Cancer 

Cells to Combinatorial siRNA Therapy 

Hamidreza Montazeri Aliabadi, Parvin Mahdipoor, Hasan Uludag. 

214 

Combination Therapy with Novel Nanoparticle, 

SNS01-T, and Bortezomib Results in Synergistic 

Cytotoxicity In Vitro and In Vivo in Multiple Myeloma 

Catherine Taylor, Qifa Zheng, Zhongda Liu, Terence Tang, Sarah 

Francis, Richard Dondero, John Thompson. 

215 

Rapamycin Enhances Adenovirus-Mediated Cancer 

Diagnosis in Naive and Pre-Immunized Hosts 

Ziyue Karen Jiang, Mai Johnson, Diana Moughon, Jennifer Kuo, 

Makoto Sato, Lily Wu. 

216 

Anti-Tumor Effects of Ad-p53 on INK4A/ARF-Defective 

Mesothelioma Are Inluenced Positively by Small G 

Protein Inhibitors but Negatively by Heat Shock Protein 

90 Inhibitors 

Masatoshi Tagawa, Kuan Chai, Yuanyuan Jiang, Kiyoko Kawamura, 

Shan Yang, Atsushi Ogino, Naoto Yamaguchi, Kazunori Kato, Yuji 

Tada, Yuichi Takiguchi, Koichiro Tatsumi, Hideaki Shimada, Kenzo 

Hiroshima, Shuji Kubo. 

217 

Lentiviral shRNA Targeting NANOG/NANOGP8 

Enhances Topotecan Chemotherapy in Human 

Colorectal Carcinomas 

John M. Jessup, Jingyu Zhang, Luis A. Espinoza, Abid Mattoo, 

Snorri S. Thorgeirsson. 

102 




4:00 PM - 6:00 PM 


Cancer-Targeted Gene & Cell Therapy: Viral 

Vector / siRNA-Mediated Therapies 

218 

Comparative Evaluation of the Antitumor Activity 

of Anti-Angiogenic Proteins Delivered by Lentivirus 

Vector in Human Malignant Mesothelioma Model 

Misato Takagi-Kimura, Tomoko Hashimoto-Tamaoki, Noriyuki 

Kasahara, Shuji Kubo. 

219 

Clinical Development of “Bifunctional” shRNA 

Knockdown of Acid Ceramidase 

James Norris, Xiang Liu, Donald D. Rao, Zhaohui Wang, Neil Senzer, 

John Nemunaitis. 

220 

Photochemical Internalization (PCI)-Enhanced 

Nonviral Gene-Directed Enzyme Prodrug Cancer 


Henry Hirschberg, Young Jik Kwon. 

221 

TRAIL-R2 and HRK Mediate Angiotensin Type 2 

Receptor-Induced Apoptosis of Human Prostate Cancer 


Nana Pei, Yanling Zhang, Feilong Jie, Jie Luo, Xinglu Chen, Jinlong 

Li, Hongwei Li. 

225 

In Vitro Repair of Rhodopsin Mutation by Spliceosome- 

Mediated RNA Trans-Splicing 

Adeline Berger, Stéphanie Lorain, Charlène Joséphine, Mélissa 

Desrosiers, Peggy Fabre, Cecile Peccate, Thomas Voit, Luis Garcia, 

José-Alain Sahel, Alexis-Pierre Bemelmans. 

226 

Muller Cell Reactivity Despite Neuronal Preservation 

after Systemic Gene Therapy 

Tonia S. Rex. 

227 

Gene Replacement Therapy for Choroideremia 

Aaron D. Black, Vidyullatha Vasireddy, Rajashekhar Gaddameedi, 

Daniel C. Chung, Albert M. Maguire, Jeannette L. Bennicelli, Tanya 

Tolmachova, Miguel C. Seabra, Jean Bennett. 

228 

Adeno-Associated Vector (Type 8)-Mediated Expression 

of siRNA Targeting Vascular Endothelial Growth Factor 

Eficiently Inhibits Neovascularization in a Murine 

Choroidal Neovascularization Model 

Tsutomu Igarashi, Koichi Miyake, Noriko Miyake, Osamu Iijima, 

Chiemi Yaguchi, Takashi Shimada, Hiroshi Takahashi. 

229 

AAV-Mediated Expression of Secreted Human Ciliary 

Neurotropic Factor (hCNTF) Leads to Long-Term 

Preservation of Cone Photoreceptors in an Autosomal 

Recessive Model of Retinitis Pigmentosa 

Daniel M. Lipinski, Alun R. Barnard, Mandeep S. Singh, Edward 

Lee, Robert E. MacLaren. 


Sensory (Ophthalmic and Auditory) Gene & 

Cell Therapy 

222 

CEP290 Directly Anchors Microtubules to the 

Membrane and Its N- and C- Termini Inhibit Protein 

Function and Ciliogenesis 

Theodore G. Drivas, Jean Bennett. 

223 

Dual Adeno-Associated Virus Vectors for Delivery of 

Large Genes to the Retina 

Frank M. Dyka, Sanford L. Boye, Shannon E. Boye, William W. 

Hauswirth. 

224 

Induced Pluripotent Stem Cells as a Model To Test Gene 

Augmentation Therapy for Choroideremia 

Vidyullatha Vasireddy, Jason A. Mills, Rajashekhar Gaddameedi, 

Etiena Basner-Tschakarjan, Monica Kohnke, Aaron H. Black, Krill 

Alexandrov, Albert M. Maguire, Daniel Chung, Helen Mac, Lisa 

Sullivan, Paul Gadue, Jeannette L. Bennicelli, Deborah L. French, 

Jean Bennett. 

230 

The Anti-Angiogenic Capacity of POMC Gene Delivery: 

From Cancer to Blindness 

Guei-Sheung Liu, Ming-Hong Tai, Gregory J. Dusting. 

231 

Genotype to Phenotype Relationship in CEP290 

Mutations: Improving Gene Therapy Strategies for the 

Most Common Form of Leber’s Congenital Amaurosis 

Adam P. Wojno, Theodore G. Drivas, Jean Bennett. 

232 

The LentiVector® Gene Therapy Platform for Ocular 

Disease: A Clinical Update 

Yatish Lad, Kyriacos Mitrophanous, Scott Ellis, James Miskin, Katie 

Binley, Michelle Kelleher, Cherry Lucas, Stuart Naylor. 


103



4:00 PM - 6:00 PM 



233 

Lentiviral Vector-Based Insertional Mutagenesis 

Identiies New Oncogenes and Molecular Networks 

That Control Hepatocyte Transformation and 

Metabolism 

Marco Ranzani, Daniela Cesana, Cynthia Bartholomae, Francesca 

Sanvito, Stefano Annunziato, Michela Riba, Fabrizio Benedicenti, 

Pierangela Gallina, Claudio Doglioni, Christof von Kalle, Yoon Jun 

Kim, Elia Stupka, Manfred Schmidt, Giovanni Tonon, Luigi Naldini, 

Eugenio Montini. 

240 

Spacio-Temporal Mutagenesis in Muscle by Using AAV- 

Cre Vectors: The Example of the Mtm1 Gene 

Romain Joubert, Alban Vignaud, Mickaël Le, Christelle Moal, Nadia 

Messaddeq, Anna Buj-Bello. 

241 

Synergistic and Tunable Gene Activation by 

Combinations of Synthetic Transcription Factors 

Pablo Perez-Pinera, David G. Ousterout, Jonathan M. Brunger, 

Alicia M. Farin, Katherine A. Glass, Farshid Guilak, Gregory E. 

Crawford, Alexander J. Hartemink, Charles A. Gersbach. 

242 

A Novel Enhancer Increases the Level and Duration of 

Transgene Expression in Mice 

Chunbo Zhang, Leping Li, Dexi Liu. 


234 

Detection of RNA Interference (RNAi) Mediated mRNA 

Cleavage in Fresh Injected Tumor Tissue from Patients 

a Phase I Trial of an Intratumoral pbi-shRNA Lipoplex 

Targeting Stathmin-1 

Zhaohui Wang, Donald D. Rao, Phillip B. Maples, Neil Senzer, John 

Nemunaitis. 

235 

LncRNA Saf Interacts with SPF45 To Promote Fas 

Receptor Alternative Splicing in Maturing Erythroid 


Olga Villamizar, Christopher B. Chambers, Janice M. Riberdy, Derek 

A. Persons, Andrew Wilber. 

236 

Stable and Selective Production of Therapeutic Protein 

in the Breastmilk of Non-Transgenic Mice Via Early 

Gestational Intra-Amniotic Gene Transfer 

Jesse D. Vrecenak, Miroslaw Kozlowski, Carlyn A. Todorow, 

Antoneta Radu, Alan W. Flake. 

237 

Improved Engraftment of Lentivirally Transduced 

Adipose Tissue-Derived Stem Cells for Hemophilia B 

Therapy Using Bioengineered Cell Sheet Technology 

Inkyong Shim, Kazuo Ohashi, Kohei Tatsumi, Natsumi Watanabe, 

Yuji Kashiwakura, Tsukasa Ohmori, Yoichi Sakata, Makoto Inoue, 

Mamoru Hasegawa, Teruo Okano. 

238 

Targeted PLGA Nanoparticles Encapsulating Histone 

Deacetylase Inhibitor, Panobinostat Effectively Control 

T Cell Leukemia 

Boyoung Choi, Jangwook Lee, Jeong-A. Shin, Dahye Lee, Kuen Yong 

Lee, Priti Kumar, Sang-Kyung Lee. 

239 

Radiosensitivity of Human Induced Pluripotent Stem 

Cells (hiPSC) Detectable by Cell Cycle Analysis 

Frank Houghton, Michael W. Epperly, Xichen Zhang, Vishwajit 

Nimgaonkar, Joel S. Greenberger. 

243 

Design and Screening of Custom Zinc Finger 

Transcription Factors To Modulate Expression of Genes 

Controlling Cell Fate 

Ashley L. Fischer, Qingzhou Ji, Erik R. Eastlund, Mark A. Gerber, 

Carol A. Kreader, Scott W. Knight. 

244 

TaqMan® Assays for Assessment of Genome, 

Epigenome and Quality of Pluripotent Stem Cells 

Andrew Fontes, Rene H. Quintanilla, Jeffrey Fergus, Uma 

Lakshmipathy. 

245 

An ‘Open-Source’ Approach to Simple and Precise 

Genome Editing with rAAV 

Rob Howes, Alex Rieck, Eric Rhodes. 

246 

UV Light Modulates Transcriptional Activity of Egr-1 

Promoter on Human Primary Tenocytes 

Francisco Martinez-F, Araceli Barrera-Lopez, Hugo Sandoval- 

Zamora, David T. Curiel, Juan A. Madinaveitia-V, Rebecca E. 

Franco-Bourland. 


247 

Embryonic Renal Progenitor Cells Activate the 

b-Catenin/TCF Signaling Pathway during Integration 

into Damaged Adult Renal Tubules 

Paul Goodyer, Zhao Zhang, Diana Iglesias. 

248 

Preclinical Investigations of Therapeutic Beneits and 

Biodistribution from Stress Erythropoiesis on Minimal 

Dose of Lysosomal Enzyme Required for Phenotypic 

Correction in Mice with Hurler Syndrome 

Jingfen Han, Mei Dai, Salim S. Elamouri, Phuong Cao, Dao Pan. 

104 




4:00 PM - 6:00 PM 



249 

Evidence of a Functional Role of Co-Culture in Delta- 

Mediated Expansion 

Korashon L. Watts, Colleen Delaney, Hans-Peter Kiem. 

250 

Rapidly Adhering Cells (RACs) Residing in Skeletal 

Muscle Contribute to Ectopic Adipose Cell Formation/ 

Accumulation in Skeletal Muscle of Dystrophin/ 

Utrophin-/- Mice 

Jihee Sohn, Aiping Lu, Ying Tang, Bing Wang, Johnny Huard. 

251 

PGE2 Increases Lentiviral Vector Transduction 

Eficiency of Human HSC 

Garrett C. Heffner, Lauryn Christiansen, Francis J. Pierciey, Jr, 

Robert Kutner, Mitchell Finer, Gabor Veres. 


253 

Amniotic Fluid Stem Cells for the Treatment of 

Hemophilia A 

Chung-Jung Kuo, Saloomeh Mokhtari, Melisa Soland, Shay Soker, 

James Yoo, John Owen, Mary Ann Knovich, Anthony Atala, Graca 

Almeida-Porada, Christopher Porada. 

254 

Characterization of Tumor Suppressive Effect Mediated 

by Human Bone Marrow-Derived Mesenchymal Stem 


Yulyana Yulyana, Ivy A. W. Ho, Jerry K. Y. Chan, Paula YP Lam. 

255 

Non-Invasive High-Resolution Imaging of Muscle 

Regeneration with a New Red-Absorbing Fluorescent 

Protein 

Jun Chu, Russell D. Haynes, Stephane Y. Corbel, Pengpeng Li, 

Emilio Gonzalez-Gonzalez, Palula J. Cranill, Michelle Baird, 

Michael W. Davidson, Christopher H. Contag, Kang Shen, Helen M. 

Blau, Michael Z. Lin. 

256 

Effects of Nano-Pillar Arrays on Differentiation of 

Human Mesenchymal Stem Cells 

Hyuk Sang Yoo, Hyesung Kim. 

257 

Integrating Non-Viral Sleeping Beauty Vector 

Delivery of Sox Transcription Factors Enhances MSC 

Chondrogenesis 

Laila Begum, Alan J. Nixon. 

258 

Variables That Affect Transduction of CD34+ 

Hematopoietic Stem Cells 

Francis J. Pierciey, Jr, Lauryn M. Christiansen, Robert Kutner, 

Garrett C. Heffner, Gabor Veres. 


259 

Assembly-Activating Protein Is Highly Tolerant To Point 

Mutations without Loss of Function: A High-Throughput 

Mutagenesis Screen Utilizing DNA Barcoding 

Lauriel Earley, Kei Adachi, Hiroyuki Nakai. 

260 

Development of a Validated Method Offering a 

Potentially Standardized Biological Assay for the 

Titration of HIV-1 Based Lentiviral Vectors 

Christian Skorik, Wesley Gorman, Mitch Finer, Mark D. Angelino, 

Robert H. Kutner. 

261 

Technology Transfer of a High Yield Fermentation 

Process and cGMP Production of Antibiotic-Free 

Plasmid DNA Vaccines 

Philippe Ledent, Olivier Geurten, Laurent Jost, Marc Daukandt, 

James A. Williams, Aaron Carnes. 

262 

Development of a Scale-Down Manufacturing Model for 

Generation of HIV-1 Based Lentiviral Vectors 

Wesley C. Gorman, Christian Skorik, Mitch Finer, Mark D. Angelino, 

Robert H. Kutner. 

263 

Antibiotic-Free Production of a Herpes Simplex Virus 2 

DNA Vaccine in a High Yield cGMP Process 

Jared Nelson, Ying Cai, Stephen Rodriguez, Neil Finlayson, James 

A. Williams, Aaron E. Carnes. 

264 

Characterization Technique for Drug and Gene Delivery 

Nanoparticles in Complex/Biological Media 

Sonja Capracotta, Duncan Grifiths, Phil Vincent, William Bernt, 

Patrick Hole. 

265 

Molecular and Cellular Analyses of Platelet-Rich 

Plasma Preparations 

Roberta S. Stilhano, Priscila M. A. Denapoli, Sheila J. M. Ingham, 

Renne J. Abdalla, Sang W. Han. 


677 

RNA-guided Genome Editing Using CRISPR-Cas Systems 

Feng Zhang 



105




4:00 PM - 6:00 PM 



678 

Rescue of T-cell deiciency in Prkdc scid mice by 

transplantation of gene-repaired haematopoietic stem 

cells 

Hayder H Abdul-Razak, Céline Rocca, Steven J Howe, María 

Eugenia Alonso-Ferrero, Richard Gabriel, Cynthia C Bartholomae, 

Chih Hao V Gan, Marina I Garín, Francisco Javier Molina Estévez, 

Alison Roberts, Michael Blundell, Guillermo Güenechea, Jianbin 

Wang, Michael C Holmes, Philip D Gregory, Christine Kinnon, 

Christof von Kalle, Manfred Schmidt, Juan Antonio Bueren, Adrian 

J Thrasher and Rafael J Yáñez-Muñoz 

679 

A microRNA-based system for selective gene expression 

in tumor-associated macrophages after adenovirus 

mediated gene transfer 

Michelle Giovani, Kamola Saydaminova, Hongjie Wang, Jonas 

Persson, Hua Cao, Erini Papapetrou, Il-Kyu Choi, Chae-Ok Yun, 

Andre Lieber 

680 

Overcoming problems in the generation of adenovirus 

vectors for genome editing in human hematopoietic 

stem cells 

Kamola Saydaminova, Xun Ye, Hongjie Wang, Akseli Hemminki, 

Eirini Papapetrou, Andre Lieber 

681 

Scalable contaminant-free production of adenoassociated 

virus vectors utilizing cytoplasmic helper 

carrier 

Biao Dong, Andrea R. Moore, Jihong Dai, Sean Roberts, Kirk Chu, 

Philipp Kapranov, Bernard Moss, Weidong Xiao 

682 

A novel RNA oligonucleotide improves liver function 

and inhibits liver carcinogenesis in vivo 

V. Reebye, P. Sætrom, P.J. Mintz, K.W. Huang, P. Swiderski, L. Peng, 

C. Liu, X.X. Liu, S. Jensen, D. Zacharoulis, N. Kostomitsopoulos, N. 

Kasahara, J.P. Nicholls, L.R. Jiao, M. Pai, M. Mizandari, T. Chikovani, 

M.M. Emara, A. Haoudi, D.A. Tomalia, J.J. Rossi, N.A. Habib 

683 

Pre-T cell receptor for improved expansion of T cells in 

the absence of TCR alpha 

Roman Galetto, Céline Lebuhotel, Laurent Poirot, Cécile Schiffer 

Mannioui, Julianne Smith, Andrew Scharenberg 

685 

Human Erythropoietin Gene Delivery for Cardiac 

Remodeling of Myocardial Infarction in Rats 

Youngsook Lee, Arlo N. McGinn, Curtis D. Olsen, Kihoon Nam, 

Minhyung Lee, Sug Kyun Shin, Sung Wan Kim 

686 

MGN1703 – structure of agonist determines cellular 

responses to TLR9 activation 

Kerstin Kapp, Christiane Kleuss, Manuel Schmidt, Matthias 

Schroff, Burghardt Wittig 


688 

Mini-LCC DNA Vector: The Gold Standard of Gene 

Delivery in Molecular Medicine 

Naiseh Naissi, Roderick Slavcev 

689 

Overcoming physical barriers in oncolytic virotherapy 

using a serotype 3-based oncolytic adenovirus 

expressing relaxin 

Joung-Woo Choi, Il-Kyu Choi, Andre Lieber, Chae-Ok Yun 

690 

Design of oncolytic HSV1 that express cellular gene 

moieties that improve antiglioma eficacy and normal 

cell safety 

Hiroshi Nakashima, Tran T. Nguyen, William F. Goins, Ennio A. 

Chiocca 

691 

Polymeric Carriers for the Delivery of Neurotrophins to 

the Brain 

Lei Miao, Devika S-Manickam, Zhijian He,Yuhang Jiang, Alexander 

V. Kabanov 

692 

A Lentiviral System for Analysis of Signaling Pathways 

Involved in Reprogramming of Induced Pluripotent 

Stem Cells 

Ashley Fritz, Sunnie Mao, David Schaffer 

693 

Pluronic block copolymers for gene delivery 

Vivek Mahajan, Alexander Kabanov 

694 

Gold nanoparticles assisted laser optoporation of cells: 

a novel physical method for eficient, safe and versatile 

gene delivery 

W. Ding, E. Bergeron, J. Baumgart, M-O. Lapointe, L. Humbert, J-J. 

Lebrun, M. Meunier 

684 

An inducible Caspase-9 delivery-system using 

Mesenchymal Stromal Cells for lung cancer treatment 

Miki Ando, Valentina Hoyos, Shigeki Yagyu, Carlos Ramos, Antonio 

Di Stasi, Gianpietro Dotti, Lisa Bouchier-Hayes, Malcolm Brenner 

695 

A potential therapeutic role for survivin in mitigating 

the harmful effects of ionizing radiation 

G Metzger, E Choi, K Carruthers, E Kocak 

106 




4:00 PM - 6:00 PM 



696 

HIV Latency: Activating Virus Expression with 

Combined Genetic and Chemical Tools 

Suresh K. Arya, PhD, Agnes Holczbauer, MD 

697 

Sensitisation of neuroblastoma cells for TRAIL-induced 

apoptosis by downregulated XIAP 

Ahmet Cingoz, Yavuz Taga, Betul Catalgol 


699 

Culture and cryopreservation of human epidermal 

melanocytes (MC) for preclinical trials 

Kim Jin Tac, Pak Seoung Hoon, Shin Back Soo, Han Su-Youne, Lee 

Hyeon Kyeong, Lee Hyun Woo, Kim Ji Hyang, Lee Ai-Young, Do 

Byung-Rok 

700 

Universal Stem Cell Gene Therapy Platform: Upgrading 

the Long-Term Gene Therapy Arm by shifting from 

Analogic to Breakthrough Digital Endonuclease- 

Boosted Gene Targeting and Focussing on Next 

Generation Ex Vivo Protocols 

Roger Bertolotti 



107


Abstract Directory, Friday, May 17, 2013 


DNA Vectorology & Gene Targeting 

1:00 PM - 3:00 PM 


266 1:00 PM 

Gene Targeting at Silent Loci with Adeno-Associated 

Virus Vectors 

Li B. Li, David R. Deyle, David W. Russell. 

267 1:15 PM 

Development of Novel Adeno-Associated Virus Variants 

Targeting HIV-Infected T Cells 

Dawn P. Wooley, John Weinstein, Poornima K. L. Narayan, Priyanka 

Sharma, David V. Schaffer, Katherine J. D. A. Excoffon. 

268 1:30 PM 

DNA Hairpins Stimulate Eficient Gene Correction Via 

Double Strand Break Repair 

Matthew L. Hirsch, Richard H. Samulski. 

269 1:45 PM 

A Transposon-Based Inducible System for Improved 

Expression In Vivo 

Joseph E. Doherty, Lauren E. Woodard, Adham S. Bear, Aaron 

Foster, Matthew H. Wilson. 

270 2:00 PM 

Minicircle DNA Is Superior to Plasmid DNA in Eliciting 

Antigen-Speciic CD8+ T Cell Responses and Generating 

Protective Immunity 

Nicole E. B. Skinner, Wynette M. Dietz, Sara E. Hamilton, Michelle 

D. Jund, Suzanne Heitfeld, Adam J. Litterman, Patrick Hwu, Zhi- 

Ying Chen, Andres M. Salazar, John R. Ohlfest, Bruce R. Blazar, 

Christopher A. Pennell, Mark J. Osborn. 

271 2:15 PM 

A Mini-Intronic Plasmid (MIP): A Novel Robust 

Transgene Expression Vector In Vivo and In Vitro 

Jiamiao Lu, Feijie Zhang, Mark A. Kay. 

272 2:30 PM 

Vertically-Integrated Translational Studies of PDX1 as 

a Therapeutic Target for Pancreatic Cancer Via a Novel 

Bifunctional RNAi Platform 

F. Charles Brunicardi, Juehua Yu, Shi-he Liu, Guisheng Zhou, Robbi 

Sanchez, Christopher M. Jay, Padmasini Kumar, Phillip B. Maples, 

Donald D. Rao, Neil Senzer, John Nemunaitis. 

273 2:45 PM 

Phase I Trial of Escalating Doses of SGT-53 Infusion in 

Combination with Docetaxel in Advanced Solid Tumor 

Patients 

John Nemunaitis, Neil Senzer, Cynthia Bedell, Derek Nemunaitis, 

Meghan Degele, Gerald Edelman, Minal Barve, Robert Nunan, 

Kathleen F. Pirollo, Antonina Rait, Esther H. Chang. 


Gene & Cell Therapy of Diabetes, Metabolic 

and Genetic Diseases I 

1:00 PM - 3:00 PM 


274 1:00 PM 

AAV Integrates Randomly into the Nuclear and 

Mitochondrial Genome after LPLD Gene Therapy 

Christine Kaeppel, Stuart Beattie, Raffaele Fronza, Richard van 

Logtenstein, Florence Salmon, Sabine Schmidt, Stephan Wolf, Ali 

Nowrouzi, Hanno Glimm, Christof von Kalle, Harald Petry, Daniel 

Gaudet, Manfred Schmidt. 

275 1:15 PM 

Ex Vivo Lentiviral Gene Therapy as a Cure for Murine 

Pompe Disease 

Merel Stok, Helen de Boer, Marshall W. Huston, Edwin H. Jacobs, 

Onno Roovers, Holger Jahr, Dirk J. Duncker, Trudi P. Visser, 

Ans T. van der Ploeg, Arnold J. J. Reuser, Nico P. van Til, Gerard 

Wagemaker. 

276 1:30 PM 

Pathogenesis of Growth Failure in Murine and Canine 

Glycogen Storage Disease-Ia and Partial Reversal of 

Growth Failure Using Adeno-Associated Viral Vector 


Elizabeth J. Brooks, Dianne Little, Ramamani Arumugam, Baodong 

Sun, Sarah Curtis, Amanda DeMaster, Michael Maranzano, Mark W. 

Jackson, Priya Kishnani, Michael S. Freemark, Dwight D. Koeberl. 

277 1:45 PM 

AAV-Mediated Direct Somatic Genetic Correction of 


Sandra Melo, Leszek Lisowski, Lisa Bashkirova, Mark A. Kay, 

Anthony E. Oro. 

278 2:00 PM 

Correction of Mitochondrial Cardiomyopathy 

Associated with Friedreich Ataxia in a Transgenic 

Model by Gene Therapy 

Brahim Belbellaa, Morgane Perdomini, Laurent Monassier, 

Laurence Reutenauer, Nadia Messaddeq, Ronald G. Crystal, Patrick 

Aubourg, Hélène Puccio. 

279 2:15 PM 

Integration Proile of Lentiviral Vectors in Gene 

Therapy for X-Adrenoleukodystrophy 

Cynthia C. Bartholomae, Nathalie Cartier, Salima Hacein-Bey- 

Abina, Ina Kutschera, Alain Fischer, Marina Cavazzana-Calvo, 

Patrick Aubourg, Manfred Schmidt, Christof von Kalle. 

280 2:30 PM 

A Partial Deiciency Model of MUT Methylmalonic 

Acidemia (MMA) Displays Diet Inducible Disease and 

Sensitivity to Acetaminophen (APAP) 

Julien S. Senac, Vijay H. Aswani, Justin R. Sysol, Irini Manoli, 

Charles P. Venditti. 

108 






1:00 PM - 3:00 PM 


281 2:45 PM 

AAV-Based over Expression of PEPCK-C Re-Patterns 

Mouse Muscle Energy Metabolism and Prevents High- 

Fat Diet Induced Obesity 

Daibang Nie, Sheri Wang, Ying Tang, Dong Wei, William Witt, Allan 

Zhao, Yifan Dai, Freddie Fu, Johnny Huard, Bing Wang. 


Neurological Therapy: Neurodegeneration 

and Imaging 

1:00 PM - 3:00 PM 

Room: 150 ABC 

287 2:15 PM 

Fusion of Either Gap-43 or Tau to the Clostridial 

Tetanus Toxin Light Chain Improves Synaptic Inhibition 

in the Rat Spinal Cord Following Adenoviral Vector 

Gene Delivery 

Eleanor M. Donnelly, Chalonda R. Handy, Marie Kim, Jeremiah 

Huang, Thais Federici, Nicholas M. Boulis. 

288 2:30 PM 

MR-Guided Delivery of AAV2-hAADC into the Midbrain 

of Macaques Results in a Safe and Eficient Axonal 

Transport of AADC into the Striatum 

Krystof S. Bankiewicz, Adrian P. Kells, Lluis Samaranch, Waldy San 

Sebastian, Nitasha Sharma, John Bringas, Phillip Pivirotto, John 

Forsayeth. 

289 2:45 PM 

Noninvasive, Quantitative PET Imaging of AAV- 

Mediated D2R80A Reporter Gene Expression in Live 

Animal Brains 

Sea Young Yoon, Datta Ponde, Harish Poptani, Jessica Bagel, 

Patricia A. O’Donnell, Charles H. Vite, John H. Wolfe. 

282 1:00 PM 

Silencing Mutant Htt by AAV-Mediated Expression of 

RNAi Ameliorates Disease Manifestations in the YAC128 

Mouse Model of Huntington’s Disease 

Lisa M. Stanek, Lamya S. Shihabuddin, Seng H. Cheng. 

283 1:15 PM 

Gene Transfer of Human Apoe e2 Alleviate the 

Progression of Amyloid Deposition In Vivo 

Eloise Hudry, Jonathan Dashkoff, Allyson Lee, Shuko Takeda, 

Robert M. Kofie, Tadafumi Hashimoto, Maria Scheel, Tara Spires- 

Jones, Michal Arbel-Ornath, Rebecca A. Betensky, Beverly L. 

Davidson, Bradley T. Hyman. 

284 1:30 PM 

Vascular Delivery of AAV9-RNAi as a Potential Therapy 

for Huntington’s Disease 

Brett Dufour, Catherine Smith, Randall Clark, Timothy Walker, Jodi 

McBride. 

285 1:45 PM 

Transcriptional Targeting of HSV Vector-Mediated 

Transgene Expression to Speciic Sensory Neuron 

Subtypes 

Mark F. Doyal, Mingdi Zhang, George Huang, Justus Cohen, Nicole 

Scheff, Michael Gold, William F. Goins, Joseph C. Glorioso. 



1:00 PM - 3:00 PM 


290 1:00 PM 

Gene Therapy Combined with NF-kappaB Inhibition for 

Duchenne Muscular Dystrophy 

Ying Tang, Robert Kang, Kara Imbrogno, Arvydas Usas, Qing Yang, 

Aiping Lu, Freddie Fu, Johnny Huard, Bing Wang. 

291 1:15 PM 

AAV Mediated Overexpression of Human a7 Integrin 

Leads to Histological and Functional Improvement in 

Dystrophic Mice 

Kristin N. Heller, Chrystal L. Montgomery, Kimberly M. Shontz, 

Paul M. L. Janssen, K. Reed Clark, Jerry R. Mendell, Louise R. 

Rodino-Klapac. 

292 1:30 PM 

Genetic Correction of Duchenne Muscular Dystrophy 

Using Zinc Finger Nucleases 

David G. Ousterout, Pablo Perez-Pinera, Pratiksha I. Thakore, 

Matthew T. Brown, Charles A. Gersbach. 


286 2:00 PM 

Mechanisms of Caspase-1 Mediated Schwannoma 

Regression 

Mehran Taherian, Shilpa Prabhakar, Giulia Fulci, Miguel Sena- 

Esteves, Xandra O. Breakeield, Gary J. Brenner. 

293 1:45 PM 

New TALENs To Correct the Reading Frame of Exon 54 

of the Dystrophin Gene 

Joel Rousseau, Jean-Paul Iyombe, Charles A. Gersbach, Jacques P. 

Tremblay. 


109





1:00 PM - 3:00 PM 


294 2:00 PM 

Restriction of Calpain3 Expression to the Skeletal 

Muscle Prevents Cardiac Toxicity and Corrects 

Pathology in a Murine Model of Limb-Girdle Muscular 

Dystrophy 

Carinne Roudaut, Florence Le Roy, Laurence Suel, Isabelle Richard. 

295 2:15 PM 

RNAi Therapy for Limb Girdle Muscular Dystrophy 

Type 1A 

Jian Liu, Lindsay Wallace, Sara Garwick-Coppens, Carol Davis, 

Chady Hakim, Susan Guckes, Michael Hauser, Jerry Mendell, Susan 

Brooks, Scott Harper. 

296 2:30 PM 

Expression of FKRP Consequently Restored Functional 

Glycosylation of a-DG in the Skeletal and Cardiac 

Muscles in FKRP Mutant Mice 

Qi Long Lu, Lei Xu, Pei Juan Lu, Chi-Hsien Wang, Elizabeth 

Keramaris, Chunping Qiao, Bin Xiao, Derek J. Blake, Xiao Xiao. 

297 2:45 PM 

Minimally Invasive Implantation of Autologous 

Chondrocytes Transduced with rAAV5-IGF-I Improves 

Longterm Cartilage Repair in Full-Thickness Chondral 

Defects in the Equine Model 

Kyla Ortved, Alan J. Nixon. 


Cancer-Targeted Gene & Cell Therapy 

1:00 PM - 3:00 PM 


298 1:00 PM 

Safety and Correlates of Clinical Response in an Early 

Phase Clinical Trial in Multiple Myeloma Patients 

Post Auto-SCT and Adoptive Immunotherapy with 

Engineered T Cells Expressing an HLA-A2 Restricted 

Afinity-Enhanced TCR for LAGE-1 and NY-ESO-1 

Bruce L. Levine, Aaron P. Rapoport, Edward A. Stadtmauer, Dan 

T. Vogl, Brendan Weiss, Gwendolyn K. Binder-Scholl, Dominic P. 

Smethurst, Jeffrey Finkelstein, Irina Kulikovskaya, Minnal Gupta, 

Erica Suppa, Joanna E. Brewer, Alan D. Bennett, Andrew B. Gerry, 

Nick J. Pumphrey, Helen K. Tayton-Martin, Daniel Williams, 

Zhaohui Zheng, Ashraf Z. Badros, Saul Yanovich, Gorgon Akpek, 

Michael Kalos, Bent K. Jakobsen, Carl H. June. 

299 Abstract Moved to Oral Abstract Session 230 

648 1:15 PM 

Neural Stem Cells Expressing a Modiied Human 

Carboxylesterase Potentiate Anti-Cancer Activity of 

Irinotecan in Murine Glioma Model 

Karen S. Aboody, Lucy Y. Ghoda, Margarita Gutova, Megan 

Gilchrist, Christine Brown, Vazgen Kankaldyyan, Elizabth Garcia, 

Lauren Hoffman, Rex A. Moats, Phil Potter, Timothy W. Synold, 

Michael Barish. 

300 1:30 PM 

Virus-Speciic Cytotoxic T Lymphocytes (CTLs) 

Redirected with a Chimeric Antigen Receptor (CAR) 

and Engineered To Respond to IL7 Resist Regulatory T 

Cell (Treg) Inhibition 

Serena K. Perna, Daria Pagliara, Aruna Mahendravada, Hao Liu, 

Malcolm K. Brenner, Barbara Savoldo, Gianpietro Dotti. 

301 1:45 PM 

Engineering Hematopoiesis for Tumor-Targeted 

Delivery of IFNa by Tumor-Iniltrating Macrophages 

Achieves Safe and Effective Inhibition of Breast 

Cancer and Its Metastases in Both Murine and Human 

Hematochimeric Models 

Giulia Escobar, Davide Moi, Anna Ranghetti, Mario L. Squadrito, 

Michele De Palma, Roberta Mazzieri, Luigi Naldini. 

302 2:00 PM 

Myeloid Suppressor Cell Blockade Potentiates Anti- 

Tumor Responses of Adoptive T Cell Transfer Therapy 

Steven Mok, Christopher Tsui, Thinle Chodon, Antoni Ribas, 

Richard C. Koya. 

303 2:15 PM 

Afinity-Enhanced Epithelial Junction Openers Improve 

Cancer Chemotherapy 

Roma Yumul, Hua Cao, Maximillian Richter, Julie Gralow, Andre 

Lieber. 

304 2:30 PM 

Glioblastoma Transfer miRNAs into Microglial Cells 

through Extracellular Vesicles 

Erik R. Abels, Kristan E. van der Vos, Johan Skog, Suzanne E. 

Hickman, Joseph El Khoury, Xandra O. Breakeield. 

305 2:45 PM 

ab-T Cell Receptor-Based Gene Therapy Targeting 

the Common Tumor-Antigen Survivin for Therapy of 

Hematological Malignancies 

Caroline Arber, Harshal Abhyankar, Helen E. Heslop, Gianpietro 

Dotti, Barbara Savoldo. 

110 




Gene Therapy for Hematologic Diseases: From 

Models to Clinical Results 

1:00 PM - 3:00 PM 


306 1:00 PM 

Lentiviral Vector-Based Gene Therapy for Wiskott- 

Aldrich Syndrome: Preliminary Results from the 

French Center 

Salima Hacein-Bey-Abina, Johanna Blondeau, Laure Caccavelli, 

Sabine Charrier, Capucine Picard, Liliane Dal-Cortivo, Guillem 

Cross, Stephane Blanche, Frederic Bushman, Nirav Malani, Alain 

Fischer, Adrian Thrasher, Anne Galy, Marina Cavazzana-Calvo. 

307 1:15 PM 

Phase II Clinical Gene Therapy Trial for Adenosine 

Deaminase-Deicient Severe Combined Immune 

Deiciency (ADA-SCID) 

Kit L. Shaw, Robert Sokolic, Suparna Mishra, Satiro de Oliveira, 

Christopher Silvin, Geiger-Schredelseker Sabine, Brown 

Berkley, Garabedian Elizabeth, Monika Smogorzewska, Denise 

A. Carbonaro, Pei-yu Fu, Alejandra Davila, Jayashree Jagadeesh, 

Michael Hershield, Alan Wayne, Neena Kapoor, Theodore Moore, 

Fabio Candotti, Donald B. Kohn. 

311 2:15 PM 

Expansion of Gene-Modiied T Cells Following 

Direct Intravenous Injection of Therapeutic Foamy 

Virus Vector in a Canine Model of Severe Combined 

Immunodeiciency 

Christopher R. Burtner, Brian C. Beard, Douglas R. Kennedy, Troy 

R. Torgerson, Andrew M. Scharenberg, David J. Rawlings, Grant D. 

Trobridge, Peter J. Felsburg, Hans-Peter Kiem. 

312 2:30 PM 

Adeno-Associated Virus-Mediated Expression of a 

C-Terminal Truncated ADAMTS13 Variant Prevents 

Shigatoxin-Induced Thrombotic Thrombocytopenic 

Purpura 

Sheng-Yu Jin, Juan Xiao, Jialing Bao, Shangzhen Zhou, Fraser J. 

Wright, X. Long Zheng. 

313 2:45 PM 

Self-Inactivating Lentiviral Vectors for Correction of 

Rag1 Severe Combined Immunodeiciency 

Karin Pike-Overzet, Anna-Sophia Wiekmeijer, Miranda R. M. Baert, 

Chantal Lagresle-Peyrou, Axel Schambach, Fang Zhang, Rob C. 

Hoeben, Salima Hacein-Bey-Abina, Arjen C. Lankester, Robbert G. 

M. Bredius, Gertjan J. A. Driessen, Adrian J. Thrasher, Christopher 

Baum, Marina Cavazzana-Calvo, Jacques J. M. van Dongen, Frank J. 

T. Staal. 

308 1:30 PM 

Lentiviral Gene Transfer for Treatment of Children 

>2 Years Old with X-Linked Severe Combined 


Suk See De Ravin, Uimook Choi, Narda Theobald, Janet Lee, 

Hongmei Wang, Xiaolin Wu, Geraldine O’Connor, Daniel W. 

McVicar, Elizabeth Kang, John T. Gray, Brian P. Sorrentino, Harry L. 

Malech. 

309 1:45 PM 

Integration Site Analysis in a Clinical Trial of Lentiviral 

Vector Based Hematopoietic Stem Cell Gene Therapy 

for Metachromatic Leukodystrophy. An 18 Months 

Follow-Up 

Eugenio Montini, Alessandra Bifi, Andrea Calabria, Luca Biasco, 

Martina Cesani, Fabrizio Benedicenti, Tiziana Plati, Simone Leo, 

Victor Neduva, Gianluigi Zanetti, Alessandro Aiuti, David Dow, 

Christof von Kalle, Manfred Schmidt, Maria Sessa, Luigi Naldini. 

310 2:00 PM 

Comparison of Integration Site Proiles between 

SCID-X1 Gene Therapy Trials Using Gammaretroviral 

Vectors with Intact or Deleted (SIN) LTRs 

Salima Hacein-Bey-Abina, Laure Caccavelli, Nirav Malani, Charles 

Berry, Frances Male, Frederic Bushman, Alain Fischer, Marina 

Cavazzana-Calvo. 



1:00 PM - 3:00 PM 


314 1:00 PM 

Pristimerin Enhances Recombinant Adeno-Associated 

Virus Serotype 2 Vector-Mediated Transgene 

Expression Both In Vitro and In Vivo 

Lina Wang, Yuan Wang, George V. Aslanidi, Arun Srivastava, 

Changquan Ling, Chen Ling. 

315 1:15 PM 

Transcriptional and Post-Transcriptional Regulation To 

Overcome Immunological Responses to Gene Therapy 

for Hemophilia A 

Stefania E. Cannizzo, Simone Merlin, Antonia Follenzi. 

316 1:30 PM 

Selective Expansion of Transduced Hepatocytes: A 

Mechanism for Modulating Transgene Levels In Vivo 

Sean C. Nygaard, Annelise Haft, Milton Finegold, Markus Grompe. 

317 1:45 PM 

Multiplex Bi-Functional Short-Hairpin RNA (shRNA) 

Targeting Single-Nucleotide KRAS Mutations in 

Pancreatic Cancer 

Donald D. Rao, Zhaohui Wang, Padmasini Kumar, Neil Senzer, John 

Nemnaitis. 



111




1:00 PM - 3:00 PM 


318 2:00 PM 

Engineering an XID-Site Speciic I-AniI Homing 

Endonuclease through Combination of Rational Design 

and Directed Evolution 

Yupeng Wang, Jordan Jarjour, Sandrine Boissel, Summer Thyme, 

Iram Khan, Joseph Pangallo, David Baker, Barry Stoddard, Andrew 

M. Scharenberg, David J. Rawlings. 

319 2:15 PM 

LMO2 Perturbs Human T Cell Development In Vivo by 

Two Different Mechanisms 

Anna-Sophia Wiekmeijer, Karin Pike-Overzet, Martijn H. Brugman, 

Willem E. Fibbe, Frank J. T. Staal. 

325 5:10 PM 

Targeting B Cell Precursor Acute Lymphoblastic 

Leukemia (ALL) with Chimeric Antigen Receptors 

(CARs) Speciic for CD19 or CD22 

Rimas J. Orentas, Waleed Haso, Daniel W. Lee, Terry J. Fry, Alan S. 

Wayne, Crystal L. Mackall. 

Poster Session II 

5:30 PM - 7:30 PM 


AAV Vectors II 

326 

Retro-Orbital Injection of RAAVs to Neonatal Mice 

Dramtically Enhances Gene Delivery to the CNS 

Jia Li, Li Zhong, Shaoyong Li, Mengxin Li, Su Qin, Ran He, Flotte R. 

Terence, Guangping Gao. 


320 2:30 PM 

Tightly Regulated Doxycycline (Dox)-Inducible 

Lentiviral Vectors for Human Myeloprotective Gene 

Therapy: In Vitro and CD34+Xenotransplant Studies 

Nico Lachmann, Roman Hilje, Sebastian Brennig, Julia Dahlmann, 

Niels Heinz, Ina Gruh, Bernd Schiedlmeier, Christopher Baum, 

Thomas Moritz. 

321 2:45 PM 

Transcription Factor Activity Analysis Using a 

Functional Multiplex Gaussia Luciferase-Based 

Reporter Assay 

Sjoerd van Rijn, Jonas Nilsson, David P. Noske, W. Peter Vandertop, 

Bakhos A. Tannous, Thomas Würdinger. 

Presidential Symposium 330 

Presidential Symposium 

3:15 PM - 5:30 PM 

Room: Ballroom E-J 

322 4:10 PM 

Kinetics and Epigenetics of Retroviral Silencing in 

Mouse Embryonic Stem Cells Deined by Deletion of a 

D4Z4 Barrier Element 

Sylvie Rival-Gervier, Mandy Lo, Shahryar Khattak, Peter Pasceri, 

Matthew Lorincz, James Ellis. 

323 4:30 PM 

Liver-Directed, Insulin-Based Gene Therapy Protects 

NOD Mice from Diabetes 

Mahzad Akbarpour, Kevin S. Goudy, Andrea Annoni, Francesca 

Sanvito, Luigi Naldini, Maria Grazia Roncarolo. 

324 4:50 PM 

On-Target Cleavage and Off-Target Activity of TALEN 

‘Nickases’ 

Thomas J. Cradick, Christopher J. Antico, Eli J. Fine, Cory R. 

Lindsay, Eric Kildebeck, David B. Roth, Matthew H. Porteus. 

327 

AAV2H22, a Vector Selected by Directed Evolution, 

Transduces More Eficiently from the Basolateral 

Surface Despite Apical Selective Pressure 

Benjamin R. Steines, David D. Dickey, Katherine J. Excoffon, David 

V. Schaffer, Joseph Zabner. 

328 

High-Eficiency Transduction of Primary Human 

Hematopoietic Stem Cells and Erythroid Lineage- 

Restricted Expression by Optimized scAAV6 Serotype 

Vectors In Vitro and in a Murine Xenograft Model In 

Vivo 

Liujiang Song, Xiaomiao Li, Giridhara R. Jayandharan, Yuan Wang, 

George V. Aslanidi, Chen Ling, Li Zhong, Guangping Gao, Mervin C. 

Yoder, Changquan Ling, Mengqun Tan, Arun Srivastava. 


330 

Comparing Dual AAV Vector Strategy Eficiencies for 

Gene Therapy In Vitro and In Vivo 

Heikki T. Turunen, María Victoria Luna Velez, Rajani Shelke, Ru 

Xiao, Jean Bennett, Luk H. Vandenberghe. 

331 

Regulation of Host Cellular DNA Binding Proteins 

Augment the Gene Transfer Eficiency of Adeno- 

Associated Virus (AAV) Vectors 

Akshaya Krishnagopal, Sangeetha Hareendran, Dwaipayan Sen, 

Arun Srivastava, Alok Srivastava, Giridhara R Jayandharan. 

332 

Differences in AAV Serotype Dependent Distribution 

and Tropism Exist within the CNS of Wild Type and 

Mutant Mice in the MPSIIIB Mouse Model 

Janine A. Gilkes, Matthew D. Bloom, Andrew R. Kolarich, Coy D. 

Heldermon. 

112 



Poster Session II, continued 

5:30 PM - 7:30 PM 


AAV Vectors II 

333 

High-Eficiency Transduction of Primary Human 

Monocyte-Derived Dendritic Cells by Recombinant 

AAV6 Vectors Containing Mutations in Surface-Exposed 

Serine and Threonine Residues 

George Aslanidi, Angela Rivers, Luis Ortiz, Jhell Pandya, Chen Ling, 

Kim Van Vliet, Mavis Agbandje-McKenna, Arun Srivastava. 

334 

Optimization of the Capsid of Recombinant Adeno- 

Associated Virus 2 Vectors: The Final Threshold 

George Aslanidi, Angela Rivers, Luis Ortiz, Chen Ling, Kim Van 

Vliet, Lakshmanan Govindasamy, Mavis Agbandje-McKenna, Arun 

Srivastava. 

335 

Directed Evolution of AAV Capsid for Targeted Cancer 


Damien Marsic, Yuan Lu, Chunxia Cao, Dane Phelan, Lakshmanan 

Govindasamy, Frederic J. Kaye, Mavis Agbandje-McKenna, Steve 

Ghivizzani, Sergei Zolotukhin. 

336 

Structures of the Retargeted AAV-DJ Vector in the 

Presence and Not of Receptor-Analog Sucrose 

Octasulfate 

Qing Xie, Nancy Meyer, Thomas F. Lerch, Dustin McCraw, Michael 

Spillman, Scott Stagg, Michael S. Chapman. 

337 

Improved AAV1 and AAV9 Vectors for Gene Delivery 

Balaji Balakrishnan, Akshaya Krishnagopal, Dwaipayan Sen, Alok 

Srivastava, Giridhara r Jayandharan. 

338 

Plasmid Integration within AAVS1 in AAV Producer Cell 

Lines 

Amy Frederick, Yuxia Luo, John Martin, Jeffery Ardinger, Donna 

Armentano, Sam Wadsworth, Karen Vincent. 

339 

Short Hairpin RNA Delivered by Adeno-Associated 

Virus Vector Eficiently Reduced Target Gene 

Expression in Mouse Primary Cortical Neurons 

Yun Lin, Holly Carlisle, Tom Wolfe, Danny Ortuno, Trish Amarante, 

Huiren Zhao, Steve Wood, Cherylene Plewa, Mike Wyszynski, Paul 

Kassner, Ki Jeong Lee. 

340 

AAV Vectors Puriied from Culture Medium with a 

Simple and Rapid Protocol Transduce Eficiently 

Murine Liver, Muscle and Retina 

Monica Doria, Antonella Ferrara, Alberto Auricchio. 


341 

Minimally Invasive Electroporation of Synthetic DNA 

Vaccines for the Induction CTL and Functional Humoral 

Immune Responses Against HIV 

Megan Wise, Karuppiah Muthumani, Kate E. Broderick, Amir S. 

Khan, Janess Mendoza, Maria Yang, Jian Yan, Natalie Hutnick, 

Niranjan Y. Sardesai, David B. Weiner. 

342 

An E3-14.7K Peptide That Promotes Microtubules- 

Mediated Transport of Plasmid DNA Increases 

Polyplexes Transfection Eficiency 

Lucie Pigeon, Cristine Gonçalves, David Gosset, Chantal Pichon, 

Patrick Midoux. 

343 

Benchtop DNA Synthesizer: Oligo-Templated 

Polymerization (OTP) 

Adi Barzel, Mark A. Kay. 

344 

Nanoparticle-Mediated Gene Delivery for Rhodopsin- 

Associated Retinitis Pigmentosa 

Zongchao Han, Rasha Makkia, Junjing Guo, Miles J. Merwin, Mark J. 

Cooper, Muna I. Naash. 

345 

Hybrid Flp-TALE and Cre-TALE Recombinases with 

Designed Target Speciicity as Genome Engineering 

Tools 

Feng Li, Riddhi Shah, Eugenia Voziyanova, Yuri Voziyanov. 

346 

Permanent Genetic Modiication of Dividing Cells Using 

Episomally Maintained S/MAR DNA Vectors and the 

Correction of a Cancer Phenotype in Renal Tumour 


Suet Ping Wong, Richard P. Harbottle. 

347 

Collagen VII Gene Delivery Via Sleeping Beauty 

Transposon in COL7A1-Deicient Keratinocytes from 

Epidermolysis Bullosa Patients 

Maria Carmela Latella, Fabienne Cocchiarella, Giandomenico 

Turchiano, Zsuzsanna Izsvak, Zoltan Ivics, Manuel Gonçalves, 

Fulvio Mavilio, Fernando Larcher, Alessandra Recchia. 

348 

DNAJB4, a Heat Shock Rotein hsp40 Homolog, Activates 

Dendritic Cells and Functions as a Molecular Adjuvant 

When Incorporated into Cancer DNA Vaccine 

Hsing-Yu Chen, Jeremy. J.W. Chen, Chi-Chen Lin. 

349 

Surface-Stabilized Calcium Phosphate Nano-Crystals 

for Eficient Gene Delivery 

Min Sang Lee, Kyuri Lee, Nak Won Kim, Ji Hoon Jeong. 



113




5:30 PM - 7:30 PM 



350 

Polymeric Micellar Nanocarriers for Gene and 

Oligonucleotide Delivery 

Kazunori Kataoka. 

351 

Methods of Facilitating Patient Participation in DNA 

Based Experimental Therapeutic Phase I Cancer 

Studies 

Wanda Strange, Cynthia Bedell, Shannon Cagnina, Jeanne Jones, 

Patricia Brown, Gladice Wallraven, John Nemunaitis. 

352 

Manufacturing Developments for Scale-Up of 

Autologous Phase II Ovarian Tumor Tissue FANG 

Vaccine 

Nicolas Taquet, John Nemunaitis, Phillip B. Maples. 



353 

Ultrasound-Guided Hepatic Injections of HDAd Vector 

Are More Eficient and Safer Than Systemic Intravenous 

Injections for Liver-Directed Gene Therapy of Crigler- 

Najjar Syndrome Type I 

Nunzia Pastore, Edoardo Nusco, Pasquale Piccolo, Sigismondo 

Castaldo, Francesco Vetrini, Donna Palmer, Libor Vitek, Philip Ng, 

Nicola Brunetti-Pierri. 

354 

Gene Therapy for Liver Diseases Using Non-Viral 

Minicircle-DNA Vector 

Hiu Man Viecelli, Richard P. Harbottle, Suet Ping Wong, Marinne 

Chuah, Thierry VandenDriessche, Cary O. Harding, Beat Thony. 

355 

Hepatic Delivery of Genes Encoding Therapeutic 

Proteins or Mirnas Using Hyperactive Transposons: A 

Platform Technology for Gene Therapy and Functional 

Genomics In Vivo 

Mario Di Matteo, Pieter De Bleser, Ermira Samara-Kuko, Marinee 

Chuah, Thierry VandenDriessche. 

356 

Liver Progenitor Cells Isolated from Adult Mice Fed a 

Choline-Deicient, Ethionine-Supplemented Diet Can 

Engraft, Expand and Mature in the Injured Liver of the 

FRG Mouse Model 

Gustavo de Alencastro, Grant J. Logan, Allison P. Dane, Sophia H. Y. 

Liao, George C. T. Yeoh, Ian E. Alexander. 

357 

Long-Term Phenotypic Correction of Hemophilia A by 

Non-Viral PiggyBac Transposon Vector 

Hideto Matsui, Mitsuhiko Sugimoto, Midori Shima, Naoko 

Fujimoto, Akitsu Hotta. 

358 

Long-Term Secretion of Arylsulfatase A without 

Antibody Formation after AAV Mediated Neonatal Gene 

Transfer into MLD Model Mice 

Noriko Miyake, Koichi Miyake, Motoko Yamamoto, Takashi 

Shimada. 

359 

In Vitro and In Vivo Characterization of an Engineered 

Arylsulfatase B for Therapy of Mucopolysaccharidosis 

VI 

Rita Ferla, Pamela Claudiani, Gabriella Cotugno, Aaron Petrey, 

Richard Steet, Alberto Auricchio. 

360 

Gene Therapy for MPS VI Is Effective in Cats without 

Pre-Existing Immunity to AAV8 

Rita Ferla, Thomas O’Malley, Roberto Calcedo, Patricia O’Donnell, 

Ping Wang, Gabriella Cotugno, Pamela Claudiani, James M. Wilson, 

Mark Haskins, Alberto Auricchio. 

361 

Correlation of Gene Transfer and Transduction to 

Chloride Current Defect from rAAV2/5 Vector Derived 

Transgene DCFTR in Murine CF Nasal Airway 

Gang Shi, Kelvin D. MacDonald, Christian Mueller, Terence R. 

Flotte, Anne C. Fischer. 

362 

Helper-Dependent Adenoviral Vectors for Liver- 

Directed Gene Therapy of Primary Hyperoxaluria Type 

1 

Raffaele Castello, Stefania D”Aria, Roberta Borzone, Pasquale 

Piccolo, Patrizia Annunziata, Nicola Brunetti-Pierri. 

363 

Fetal Gene Therapy at 0.4G with AAV-hFIX or AAV-hFX 

Mediates Therapeutic Transgene Expression Despite 

Growth and a Positive Immune Response in a Non- 

Human Primate Model 

Citra Mattar, Cecilia Rosales, Rowell Fragata, Nuryanti Johana, 

Simon Waddington, Arijit Biswas, Mahesh Choolani, Amit 

Nathwani, Jerry Chan. 

364 

AAV Vector-Mediated Iduronidase Gene Delivery in 

a Murine Model of Mucopolysaccharidosis Type I: 

Comparing Different Routes of Delivery to the CNS 

Lalitha Belur, Zhenhong Nan, Kelly M. Podetz-Pedersen, Carolyn 

A. Fairbanks, Leah R. Hanson, William H. Frey, Karen Kozarsky, 

Walter C. Low, R. Scott McIvor. 

114 




5:30 PM - 7:30 PM 




365 

Enzyme Replacement Therapy Enhanced a Therapeutic 

Eficacy of Bone Marrow Transplantation in MPS II 

Mouse 

Kazumasa Akiyama, Yohta Shimada, Takashi Higuchi, Sayoko 

Iizuka, Makoto Otsu, Hiromitsu Nakauchi, Jillian R. Brown, Brett 

E. Crawford, Takahiro Fukuda, Hiroshi Kobayashi, Yoshikatsu Eto, 

Hiroyuki Ida, Toya Ohashi. 


366 

Ex-Vivo Transduced Monocytes as Gene Delivery 

Vehicles to Restenotic Arteries 

Ilia Fishbein, Richard F. Adamo, Jeanne M. Connolly, Shira Redlich, 

Katherine A. Clark, Ricardo A. Corrales, Michael Chorny, Scott P. 

Forbes, Robert J. Levy. 

367 

AAV9-Mediated miR-499 Expression Is Suficient To 

Induce Progressive Heart Failure in Mice 

Ru-Hang Tang, Bin Xiao, Zhenhua Yuan, Chunping Qiao, Jianbin Li, 

Bo He, Juan Li, Xiao Xiao. 

368 

Eficacy of Various Populations of Murine Mesenchymal 

Stem Cells for the Treatment of Critical Limb Ischemia 

Francesca Bortolotti, Laura Ukovich, Vahid Razban, Valentina 

Martinelli, Giulia Ruozi, Franca Dore, Mauro Giacca, Serena 

Zacchigna. 

369 

Functional Impairment of Circulating Angiogenic Cells 

for Treatment of Myocardial Infarction Is Differentially 

Driven by Both Advanced Age and Coronary Artery 

Disease, and Is Improved or Further Impaired by 

Modulating Nitric Oxide Synthase Expression 

Qiumei Chen, Monika Varga, Xiaoyin Wang, Daniel J. Haddad, 

Songtao An, Lejla Medzikovic, Yan Zhang, Brian T. Clifford, Wenhui 

Gong, Jeffrey R. Fineman, Christian Heiss, Kirstin A. Aschbacher, 

Yerem Yeghiazarians, Matthew L. Springer. 

370 

Dobutamine Stress Enhances Echocardiography 

Detection in mdx Mouse Heart and Reveals Therapeutic 

Beneits by Minidystrophin Gene Therapy 

Chunping Qiao, Chunxia Zhao, Chi-Hsien Wang, Zhenhua Yuan, 

Jianbin Li, Juan Li, Xiao Xiao. 

371 

Ixmyelocel T: A Cellular Therapy for the Treatment of 

Severe, Chronic Cardiovascular Disease 

Ronnda L. Bartel. 

372 

Prevention of Restenosis through Ad-iNOS Gene 

Therapy Combined with Supplementation of 

Tetrahydrobiopterin and Arginine 

Scott P. Forbes, Ivan Alferiev, Richard F. Adamo, Michael Chorny, 

Robert J. Levy, Ilia Fishbein. 

373 

Safety of Direct Administration of AdVEGF-All6A+, an 

Adenovirus Vector Expressing a cDNA/Genomic Hybrid 

of Human Vascular Endothelial Growth Factors, to the 

Ischemic Myocardium of Rats 

Stephen M. Kaminsky, Lucy Quach, Stacey Chen, Lorraine Pierre- 

Destine, Benjamin Van de Graaf, Sébastien Monette, Jonathan B. 

Rosenberg, Dolan Sondhi, Neil R. Hackett, Ronald G. Crystal. 

374 

A Novel Anti-Inlammatory Formulation Approach 

To Enhance Myocardial Gene Transfer: An In-Vitro 

Assessment of Aspirin Loaded Poly-Lactic Glycolic Acid 

Nanoparticles 

Anthony S. Fargnoli, Anbin Mu, Michael G. Katz, Richard D. 

Williams, Marina V. Sumaroka, Robert Levy, David B. Weiner, Scott 

L. Diamond, Kenneth B. Margulies, Shu Yang, Charles R. Bridges. 

375 

Development of In Vitro and In Vivo Evaluation 

Methods for Gene Therapy Products 

Il Ung Oh, Ki Dae Park, Soyeong Kang, Ju Hyun Choi, Sung Yeol 

Han, Min Jeong Choi, Sun-Young Baek. 


376 

Restoration of the Motor and Behavior Phenotype 

with AAV9 in Aromatic L-Amino Acid Decarboxylase 

Deiciency Mice 

Ni-Chung Lee, Yin-Hsiu Chien, Shin-ichi Muramatsu, Barry J. 

Byrne, Wuh-Liang Hwu. 

377 

Signiicant Therapeutic Beneit after Post-Symptomatic 

Gene Therapy in a Feline Model of GM2 Gangliosidosis 

Victoria J. McCurdy, Ashley N. Randle, Heather L. Gray-Edwards, 

Allison M. Bradbury, Aime K. Johnson, Patricia M. Beadlescomb, 

Miguel Sena-Esteves, Douglas R. Martin. 

378 

Tight Regulation of Transgene Expression by 

Mifepristone-Inducible System after In Vitro Delivery 

by Lentiviral Vectors to Neural Progenitor Cells 

Christopher Grunseich, Yijun Shi, Michael Mooney, Kenneth H. 

Fischbeck, Tyler M. Pierson. 



115




5:30 PM - 7:30 PM 



379 

Intravenous AAV9 Based Gene Therapy Corrects a 

Mouse Model of Sandhoff Disease 

Jagdeep S. Walia, Alexander Bello, Naderah Altaleb, Christa Kruck, 

Richard Hemming, Gary P. Kobinger, Barbara Triggs-Raine. 

380 

A Speciic Cleavage of Amyloid-b by a Viral Protease 

NIa Prevents the Amyloid-b-Mediated Mitochondrial 

Dysfunction 

Woo Jin Park, Bae-Hyun Shin, Hye-Eun Han, Sang Min Park. 

381 

Viral Vector Mediated Overexpression of Pleiotrophin 

for Disease Modiication in Parkinson’s Disease 

S. E. Gombash, F. P. Manfredsson, C. J. Kemp, S. L. Wohlgenant, N. C 

Kuhn, S. M. Fleming, R. J. Mandel, T. H. Collier, C. E. Sortwell. 

382 

Infrared Fluorescent Protein (iRFP) as a Reporter for 

Monitoring and Modulating Neurons 

Christopher T. Richie, Lowella V. Fortuno, Douglas B. Howard, 

Leslie R. Whitaker, Bruce T. Hope, Brandon K. Harvey. 

383 

Fetal Brain-Directed AAV5 and AAV9 Gene Therapy 

Results in Rapid, Robust, and Persistent Transgene 

Expression in Mouse Choroid Plexus Epithelia 

Marie Reine Haddad, Eun-Young Choi, Anthony Donsante, Patricia 

M. Zerfas, Stephen G. Kaler. 

384 

Intravenous Administration of AAV9-mbgal in a GM1 

Gangliosidosis Mouse Model Expresses Enzyme in the 

CNS and Delays Disease Onset with Gender Differences 

Cara M. Weismann, Jennifer Ferreira, Stacy Maitland, Quin Su, 

Guangping Gao, Miguel Sena-Esteves. 

385 

Gene Delivery of ATP6 by a Mitochondrial Targeting 

Sequence Modiication of AAV9 Capsid VP2 Rescues 

Cells with Mutated T8993G MtDNA Responsible for 

Neuropathy Ataxia and Retinitis Pigmentosa and 

Expresses in the Mouse CNS 

John Guy, Huijan Yuan. 

386 

rAAV2/5-Mediated Gene Transfer Is Less Eficient 

in the Aged Rat Brain: Examination of a-Synuclein- 

Mediated Neurodegeneration in the Nigrostriatal 

System 

Sara E. Gombash, Fredric P. Manfredsson, Christopher H. Kemp, 

David L. Fischer, Nathan C. Kuhn, Allyson Cole-Strauss, Ronald J. 

Mandel, Sheila M. Fleminig, Timothy J. Collier, Jack W. Lipton, Caryl 

E. Sortwell. 

387 

Safety Study of Intracranial AAVrh8-Mediated Gene 

Transfer of beta-Hexosaminidase in Non-Human 

Primates 

Diane Golebiowski, Imramsjah M. J. van der Bom, Churl-Su Kwon, 

Stacy Maitland, Anna Luisa Kühn, Nina Bishop, Elizabeth Curren, 

Nilsa Silva, Andrew Miller, Susan Westmoreland, Matthew Gounis, 

Douglas R. Martin, Wael Asaad, Miguel Sena-Esteves. 

388 

Targeting Human SOD1 Using AAV Mediated RNAi in a 

Mouse Model of Amyotrophic Lateral Sclerosis 

Lorelei I. Stoica, Gabriella Toro, Robert Brown, Christian Mueller, 

Miguel Sena-Esteves. 

389 

TALEFrat#8–VP64 Increases Frataxin Gene and Protein 

Expression in Fibroblasts of Friedreich Patients and of 

Mouse Model 

Jacques P. Tremblay, Pierre Chapdelaine, Jean-Paul Iyombe, Joël 

Rousseau, Zoé Coulombe. 

390 

Transduction of the Central Nervous System Following 

Intra-Cerebroventricular Administration of Adeno- 

Associated Viral Vectors to Neonatal and Juvenile Mice 

Shervin Gholizadeh, Sujeenthar Tharmalingam, Margarita E. 

MacAldaz, David R. Hampson. 

391 

Serial and Endpoint Biomarkers for Cardiorespiratory 

Function in a Canine Model of DMD Provide an 

Integrative View of Disease Pathogenesis and Response 

to Therapy 

Andrew Mead, Alock Malik, Mihail Petrov, Martin Childers, Janet 

Bogan, Benjamin Kozyak, Hansell Stedman. 

590 (Moved from Poster Session III) 

Intracranial AAV Gene Therapy Extends the Life Span 

of GM2 Gangliosidosis Cats >Four-Fold with No Clinical 

Evidence of Vector Toxicity 

Allison M. Bradbury, Victoria J. McCurdy, Aime K. Johnson, Heather 

Gray-Edwards, Brandon L. Brunson, Ashley N. Randle, Nancy R. 

Cox, Miguel Sena-Esteves, Douglas R. Martin. 

116 




5:30 PM - 7:30 PM 


Cancer Immunotherapy II 

392 

Restored Expression of Heparanase in Tumor 

Speciic T Cells Enhances Their Antitumor Effects in 

Neuroblastoma 

Ignazio Caruana, Valentina Hoyos, Gerrit Weber, Barbara Savoldo, 

Dario Marchetti, Gianpietro Dotti. 

393 

Single-Cell Imaging Reveals That Subsets of T Cells 

Expressing a CD19-Speciic Chimeric Antigen Receptor 

Differ in Effector Function 

Harjeet Singh, Ivan Liadi, Gabrielle Romain, Navin Varadarajan, 

Laurence J. N. Cooper. 

394 

MCSP-Chimeric Antigen Receptor (CAR)-Redirected T 

Cells Target Multiple Solid Tumors 

Claudia Arambula, Barbara Savoldo, Valentina Hoyos, Eric Yvon, 

Michele Del Vecchio, Soldano Ferrone, Gianpietro Dotti. 

395 

Tumor Recurrences Share Immunogenic Antigens 

across Both Tumor Types and Primary Treatments 

Which Can Be Therapeutically Targeted with VSV-cDNA 

Libraries 

Nicolas Boisgerault, Jose Pulido, Timothy Kottke, Oliver Donnelly, 

Esteban Celis, Jill Thompson, Rosa Diaz, Kevin Harrington, Hardev 

Pandha, Peter Selby, Alan Melcher, Richard Vile. 

396 

BARF1-Speciic T Cells for the Adoptive Immunotherapy 

of EBV-Positive Nasopharyngeal Carcinoma 

Mamta Kalra, Minhtran C. Ngo, Ulrike Gerdemann, Ann Leen, 

Chrystal Louis, Cliona M. Rooney, Stephen Gottschalk. 

397 

IL-12 Increases Immune Responses Induced by a Novel 

pDNA Prostate Cancer Immunotherapy Approach in 

Non-Human Primates 

Bernadette Ferraro, Amritha Balakrishnan, Jewell N. Walters, 

Devin J. Myles, Jian Yan, Amir S. Khan, Niranjan Y. Sardesai, David 

B. Weiner. 

398 

Transgenic Expression of a Novel Immunosuppressive 

Signal Converter on T Cells 

Norihiro Watanabe, Usanarat Anurathapan, Malcolm K. Brenner, 

Helen E. Heslop, Ann M. Leen, Cliona M. Rooney, Juan F. Vera. 

399 

Hypomethylating Agent 5-aza Cytidine Exposure To 

Improve AP1903 Treatment for Apoptosis Induction of 

iCasp9/DCD19 Gene Modiied T Cells 

Elodie Bole-Richard, Jean-Marie Certoux, Idir Idirene, Laurent 

Jossot, Eric Deconinck, Fabrice Larosa, Etienne Daguindau, 

Christophe Borg, Pierre Tiberghien, Christophe Ferrand, Marina 

Deschamps. 

400 

Adapting the Spontaneous Canine Osteosarcoma Model 

for T-Cell Therapy 

Melinda Mata, Sunitha Kakarla, Juan Vera, Lisa Wang, Cliona 

Rooney, Heather Wilson, Stephen Gottschalk. 

401 

T-Cell Engineering for Adoptive Immunotherapy Using 

TAL-Effector Nucleases (TALENTM) 

Laurent Poirot Poirot, Cécile Schiffer-Mannioui, Agnes Gouble, 

Roman Galetto, Stephanie Grosse, Isabelle Chion-Sotinel, Diane Le 

Clerre, Laetitia Lemaire, Céline Lebuhotel, Justin Eyquem, Sylvain 

Arnould, Julianne Smith, Andrew Scharenberg, Sophie Derniame. 

402 

Randomized Phase II Trial of Post-Operative Adjuvant 

Chemotherapy ±FANG Autologous Tumor Cell Vaccine 

in Colorectal Carcinoma with Liver Metastases 

Joseph Kuhn, Neil Senzer, Minal Barve, G. Thomas Shires, 

Padmasini Kumar, Donald D. Rao, Gladice Wallraven, Beena O. 

Pappen, Phillip B. Maples, John Nemunaitis. 

403 

The Potential of Pro-Apoptotic PAC-1 Treatment 

for Augmentation of IL-2 Mediated Breast Cancer 

Immunotherapy 

Shyambabu Chaurasiya, Paggy Hew, Paul J. Hergenrother, David 

Sharon, Mary Hitt. 

404 

Effect of IL-15 Gene Therapy and mTOR Inhibitor 

Everolimus on Experimental Metastatic Breast Cancer 

Growth 

Na Zhao, Xiaodan Li, Feng Qi, Yujie Qiu, Xianghui He, Liwei Zhu. 


405 

Measles Virus for Immunovirotherapy of Cancer 

Christian Grossardt, Christine E. Engeland, Sascha Bossow, Silke 

Hamzaoui-Nord, Niels Halama, Dirk Jäger, Christof von Kalle, Guy 

Ungerechts. 

406 

Oncolytic Herpes Simplex Viruses Encoding an 

Antibody Retargeted C5a Complement Protein for 

Multiple Myeloma Therapy 

Hongtao Li, Stephen J. Russell. 


407 

Mengovirus Therapy for Multiple Myeloma 

Autumn Ruiz, Elizabeth M. Hadac, Stephen J. Russell. 


117




5:30 PM - 7:30 PM 



408 

Systemic Administration of Fiber Redesigned 

Infectivity-Selective Oncolytic Adenovirus for Cancer 

Treatment 

Yoshiaki Miura, Joohee Han, Julia Davydova, Masato Yamamoto. 

409 

Therapeutic Eficacy of Oncolytic Herpes Virus and 

Chemotherapy for Treatment of Late-Stage Ovarian 

Cancer 

Chelsea M. Bolyard, Balveen Kaur. 

410 

T-Cell Engager-Armed Oncolytic Vaccinia Virus 

Signiicantly Enhances Antitumor Therapy 

Feng Yu, Stephen M. Gottschalk, Xiao-Tong Song. 

411 

Improving the Oncolytic Activity of Measles Virus 

by Enhancing the Neutrophil-Mediated Response to 

Infected Tumor Cells 

Aditi Dey, Yu Zhang, Anna Z. Castleton, Bella Patel. 

412 

Directed Evolution of Oncolytic HSV To Overcome 

Resistance to Virotherapy Due to Low Entry Receptor 

Expression 

Pin-Yi Wang, Pearlly Yan, Ralf Bundschuh, Hok-Hei Tam, Timothy 

P. Cripe. 

413 

VSV Pseudotyped with Maraba Virus G Glycoprotein 

Escapes Serum Neutralization 

Mulu Z. Tesfay, Arun Ammayappan, Mark J. Federspiel, Glen N. 

Barber, David F. Stojdl, Kah-Whye Peng, Stephen J. Russell. 

414 

Oncolytic Adenovirus-Mediated Interferon 

Expression Augments Chemoradiation Therapy in 

Immunocompetent Pancreatic Cancer Model 

Joohee Han, Yoshi Miura, Ryan Shanley, Xianghua Luo, Kazunori 

Aoki, Selwyn M. Vickers, Masato Yamamoto, Julia Davydova. 

415 

High-Resolution SPECT/CT Imaging Elucidates 

Molecular Determinants of Viral Oncolysis in Primary 

and Metastatic Lymphoma 

Tanner S. Miest, Marie Frenzke, Roberto Cattaneo. 

416 

Enhanced GBM Therapy Using a New Class of HSV 

Oncolytic Vector 

Lucia Mazzacurati, Marco Marzulli, Bonnie Reinhart, Justus B. 

Cohen, Glorioso C. Joseph, Grandi Paola. 

417 

Gene Expression Proiling Identiies Predictive Markers 

of Response to Oncolytic Measles Virus Therapy 

Ianko Iankov, Cory Allen, Ian Parney, Caterina Giannini, Evanthia 

Galanis. 

Hematologic and Immunologic Gene & Cell 

Therapy I 

418 

Enhanced Engraftment of Hematopoietic Stem 

Cells (HSCs) by Inhibition of Mammalian Target of 

Rapamycin 

Emily A. Partridge, Jesse Vrecenak, Merek Kozlowski, Alan W. 

Flake. 

419 

Improved Expansion of Primitive Erythroid/ 

Megakaryocyte Precursors from Cord Blood CD34+ 

Cells with Hyperactive Variants of Pharmacologically- 

Regulated Mpl 

Eyayu Belay, Christophre P. Miller, Amanda Kortum, Beverly Torok- 

Storb, C. Anthony Blau, David W. Emery. 

420 

The Challenge of HSCs Procurement for Gene 

Therapy of beta-Thalassemia: Exploring Plerixafor as 

Mobilization Agent 

Maria Rosa Lidonnici, Annamaria Aprile, Marta Frittoli, Giacomo 

Mandelli, Bernhard Gentner, Laura Bellio, Elena Cassinerio, Laura 

Zanaboni, Silvano Rossini, Maria Domenica Cappellini, Fabio 

Ciceri, Sarah Marktel, Giuliana Ferrari. 

421 

Leukocyte Telomere Maintenance after Transplant and 

In Vivo Chemoselection of Mutant MGMTP140K Gene- 

Modiied Hematopoietic Cells 

Christopher R. Burtner, Devikha Chandrasekaran, Brian C. Beard, 

Hans-Peter Kiem, Jennifer E. Adair. 

422 

Assessing Clonal Composition of Human Hematopoiesis 

in an NSG Transplantation Model after In Vitro 

Expansion of Transduced Cord Blood CD34 Cells 

Reinhard Haemmerle, Ruhi Phaltane, Michael Rothe, Thomas 

Moritz, Ute Modlich. 

423 

Targeted Expansion of Individual Hematopoietic 

Lineages by Regulated Mpl Signaling 

David W. Emery, Eyayu Belay, Christopher P. Miller, Beverly Torok- 

Storb, C. Anthony Blau. 

424 

Correction of Murine Sickle Cell Disease Using Non- 

Ablative Conditioning and Autologous Bone Marrow 

Cells Transduced with a Gamma Globin Lentiviral 

Vector 

Tamara I. Pestina, Phillip W. Hargrove, Hui Fen Zhao, Derek A. 

Persons. 

118 




5:30 PM - 7:30 PM 



Therapy I 

425 

Evaluating a LM-PCR and Pyrosequencing Method 

for the Detection of Clonal Contribution in Polyclonal 

Retrovirally Transduced Samples 

Martijn H. Brugman, Julia D. Suerth, Michael Rothe, Sebastian 

Suerbaum, Axel Schambach, Ute Modlich, Olga S. Kustikova, 

Christopher Baum. 

426 

Disease Modeling Using Induced Pluripotent Stem Cells 

Derived from Severe Congenital Neutropenia Patients 

Lisa Trump, Ramesh Nayak, Jose Cancelas, Carolyn Lutzko. 

427 

Enhanced Engraftment of Murine and Human 

Hematopoietic Stem Cells Mobilized Using the 

Plerixafor+G-CSF Combination 

Nikoleta Psatha, Garyfalia Karponi, Eleni Sgouramali, Athanasios 

Siametis, Panayotis Baliakas, Isabelle Riviere, Michel Sadelain, 

Achilles Anagnostopoulos, George Stamatoyannopoulos, Thalia 

Papayannopoulou, Evangelia Yannaki. 

428 

Evaluation of Engraftment and Tolerance Induction 

after Reduced Intensity Conditioning for Hematopoietic 

Stem Cell Transplantation in the Rhesus Gene Therapy 

Model 

Naoya Uchida, Molly Evans, Richard Weitzel, Aylin Bonifacino, 

Sandra Price, Allen Krouse, Mark Metzger, Matthew Hsieh, Robert 

Donahue, John Tisdale. 

429 

Myeloprotective Properties of Inducible (shRNA- 

Mediated) Knock-Down of Deoxycytidine-Kinase (dCK) 

in Human Hematopoietic In Vitro Models 

Nico Lachmann, Kevin Czarnecki, Miriam Hetzel, Sebastian 

Brennig, Niels Heinz, Bernd Schiedlmeier, Thomas Moritz. 

430 

Comparision of In-Utero Gene Therapy and In-Utero 

Hemopoietic Stem Cell Therapy Approaches in Severe 

Models of Murine beta Thalassemia 

Niraja Dighe, Citra Mattar, Lay Geok Tan, Daren Sandikin, Simon 

Waddington, Michael Antoniou, Mahesh Choolani, Jerry Chan. 

431 

Regulatory T Cells Suitable for Clinical Use Can Be 

Propagated on Artiicial Antigen Presenting Cells 

Pappanaicken Kumaresan, Chetan Dhamne, Sourindra Maiti, 

Janani Krishnamoorthy, Radhika Thokala, Helen Huls, Champlin 

Richard, Dean Lee, Dat Tran, Laurence Cooper. 

Gene and Cell Therapy for Pulmonary 

Diseases 


433 

Eficient Gene Delivery to Pig Airway Epithelia 

and Submucosal Glands Using Helper-Dependent 

Adenoviral Vectors 

Huibi Cao, Tiago N. Machuca, Jonathan Yeung, Jing Wu, Kai Du, 

Cathleen Duan, Riccardo Bonato, Allan L. Coates, Kitty Leung, 

Herman Yeger, Ernest Cutz, Mingyao Liu, Shaf Keshavjee, Jim Hu. 

434 

Minicircle Plasmids Result in Enhanced and Prolonged 

Transgene Expression of Airway Epithelial Cells Both In 

Vitro and In Vivo 

Mustafa M. Munye, Rachel E. Brown, Aristides D. Tagalakis, 

Josephine L. Barnes, Rupinder K. Ghatrora, Martin Pule, Mark F. 

Lythgoe, Robin J. McAnulty, Steven J. Howe, Stephen L. Hart. 

435 

Novel Human Bronchial Epithelial Cell Lines for the 

Study of Gene Therapy Approaches To Treat Primary 

Ciliary Dyskinesia 

Mustafa M. Munye, Claire M. Smith, Rob A. Hirst, Amelia Shoemark, 

Juliette M. Delhove, Tristan R. Mckay, Claire Hogg, Christopher 

O’Callaghan, Steven J. Howe, Stephen L. Hart. 

436 

Upregulation of a Cluster of microRNAs on Mouse 

Chromosome 12qF1 in a Kras Mutant Mouse Model of 

Lung Adenocarcinoma 

Paul N. Valdmanis, Biswajoy Roy-Chaudhuri, Hak Kyun Kim, 

Leanne C. Sayles, Yanyan Zheng, Chen-Hua Chuang, Debbie 

Caswell, Monte Winslow, E. A. Sweet-Cordero, Mark A. Kay. 

437 

Lentiviral-Mediated Functional Correction of 

Hermansky-Pudlak Syndrome Type 1 Melanocytes 

Yasuhiro Ikawa, Richard Hess, Heidi Dorward, Bernadette 

Gochuico, William A. Gahl, Fabio Candotti. 

438 

Development of a Myeloid Cell-Binding Adenovirus for 

the Potential Gene Therapy of Alpha-1 Anti-Trypsin 

Deiciency 

Maurizio Buggio, Christopher Towe, Michael O. Alberti, Justin C. 

Roth, Anand C. Annan, David T. Curiel. 

439 

MicroRNAs as Potential Targets for Therapy of 

Pulmonary Hypertension 

Igor B. Gubrij, Amanda K. Pangle, Li Pang, Larry G. Johnson. 

440 

CVB3 Infection Elicits Potent Oncolytic Activity Against 

Lung Cancer Stem Cells 

Miyako Sagara, Hiroyuki Inoue, Shohei Miyamoto, Chika Sakamoto, 

Yuki Nakano, Koichi Takayama, Hiroyuki Shimizu, Yoichi 

Nakanishi, Kenzaburo Tani. 



119




5:30 PM - 7:30 PM 


Immunologic & Host Responses in Gene & Cell 


441 

Liver-Directed Gene Therapy with Lentiviral Vector 

Reverts Pre-Existing Immunity to Coagulation Factor IX 

and Rescues Hemophilia B Mice from the Disease 

Andrea Annoni, Alessio Cantore, Kevin Goudy, Mahzad Akbarpour, 

Fabio Russo, Sara Bartolaccini, Luigi Naldini, Maria Grazia 

Roncarolo. 

442 

Combination Use of TLR7 Ligand with GM-CSF Gene- 

Transduced Tumor Vaccines Provides Substantial 

Antitumor Immunity Against Poorly Immunogenic 

Mouse Lung Cancer Cells 

Megumi Narusawa, Hiroyuki Inoue, Chika Sakamoto, Tomoko 

Inoue, Shohei Miyamoto, Makoto Inoue, Koichi Takayama, Mamoru 

Hasegawa, Yoichi Nakanishi, Kenzaburo Tani. 

443 

Elimination of Contaminating Capsid-Expressing Genes 

in AAV Vectors Reduces Immune Responses to Vector 

Administration in a Canine Gene Therapy Model 

Zejing Wang, Christine L. Halbert, Donghoon Lee, Tiffany Butts, 

Rainer Storb, Stephen H. Tapscott, Dusty Miller. 

444 

Immune Activation and Transgene Stability Following 

AAV9 Gene Transfer to an Acute Myocardial Infarct 

Mouse Model 

Susan Faust, Ehre Gao, Rebecca Tenney, Jason Ohlstein, Peter Bell, 

Walter Koch, James Wilson, Joseph Rabinowitz. 

445 

Blood Proteins Affecting rAAV Eficiency 

Jerome Denard, Hans Jörg Fehling, Philippe Moullier, Tomas Voit, 

Fedor Svinartchouk. 

446 

B & T Cell Immunomodulation Attenuates Anaphylaxis 

in a Novel Mouse Model of Pompe Disease 

Phillip A. Doerler, Sushrusha Nayak, Roland W. Herzog, Barry J. 

Byrne. 

447 

Quantiication of Alpha-L-Iduronidase Expression 

Outcomes in Mice Following Sleeping Beauty 

Transposon-Mediated Gene Therapy 

Elena L. Aronovich, Bryan C. Hall, Jason B. Bell, R. Scott McIvor, 

Perry B. Hackett. 

449 

Over-Expression of Interleukin-6 Alone Induces 

Dexamethasone-Relieved Multiple-Organ Lesion in 

Mice 

Tao Bian, Zhenhua Yuan, Chunxia Zhao, Ting Zhang, Juan Li, Xiao 

Xiao. 


450 

AAV6-Mediated Systemic Administration of miR30- 

Based RNAi Hairpins Impacts Muscle Disease in the 

HSALR Myotonic Dystrophy Mouse Model 

Darren R. Bisset, Maja Zavaljevski, Carrie Stoltzman, Joel R. 

Chamberlain. 

451 

Peptide-Conjugated Morpholino Rescues Utrophin- 

Dystrophin-Deicient Mice by Restoring Dystrophin 

Expression and Improvement in Muscle Pathology and 

Function 

Bo Wu, Caryn Cloer, Stephanie Milazi, Mona Shaban, Sapana N. 

Shah, Peijuan Lu, Hong M. Moulton, Qi Long Lu. 

452 

Co-Delivery of VEGF siRNA and IL-4 into Chitosan 

Nanoparticles in Breast Tumor Model of Rat 

Emine Salva, Suna Özbas-Turan, Levent Kabasakal, Fatih Eren, 

Saadet Alan, Naziye Özkan, Jülide Akbuga. 

453 

Systemic Delivery of a Peptide-Linked Morpholino 

Oligonucleotide Neutralizes Mutant RNA Toxicity in a 

Mouse Model of Myotonic Dystrophy 

Andrew J. Leger, Leocadia M. Mosquea, Nicholas P. Clayton, 

I.-Huan Wu, Timothy Weeden, Carol A. Nelson, Lucy Phillips, 

Errin Roberts, Peter A. Piepenhagen, Seng H. Cheng, Bruce M. 

Wentworth. 

454 

Gene Expression from Pseudouridine and 

5-Methylcytidine Modiied Messenger RNAs 

Jiehua Zhou, Maggie L. Bobbin, Julie Escamilla-Powers, Eric 

Arnold, Anthony Lauer, Laura Juckem, Karl-Dimiter Bissig, Anton 

P. McCaffrey, John J. Rossi. 

455 

Integrin-Targeted Nanoparticle Delivery of miRNA for 

Anti-Tumor Therapy 

Puthupparampil Scaria, Yijia Liu, Betty Zhou, Martin Woodle. 

456 

Multi-Subtype Targeting, Escape-Reducing (MuSTER) 

Anti-HIV shRNA Gene Therapy 

Ulrike Jung, John J. Rossi. 

448 

Hepatocyte Heparan Sulfate Is Dispensable for 

Adenovirus Liver Transduction In Vivo 

Anne K. Zaiss, Erin Foley, Ding Xu, Roger Lawrence, Jeffrey D. Esko, 

Harvey R. Herschman. 

120 




5:30 PM - 7:30 PM 



457 

RNA Interference-Based Gene Therapy for Parkinson’s 

Disease Using shRNA That Speciically Targeting the 

LRRK2 G2019S Allele 

Liang Huang, Xiaomin Su, Juan Wang, Kathleen Maguire-Zeiss, 

Howard J. Federoff. 

458 

An RNAi Screening Platform To Identify Modulators of 

Glioma Stem Cells Viability and Differentiation 

Christian E. Badr, Romain Amante, Stephanie Van Hoppe, Bakhos 

A. Tannous. 

459 

Short Hairpin RNAs (shRNA) Against Steroid Receptor 

Coactivator-3/Ampliied in Breast Cancer 1 (SRC-3) 

and Steroid Receptor Coactivator-1 (SRC-1) as Breast 

Cancer Therapeutic Agents 

David M. Lonard, Donald Rao, John J. Nemunaitis, Neil N. Senzer, 

Bert W. O’Malley. 

460 

Lentiviral-Mediated ENaCa Knockdown, as a Treatment 

for Cystic Fibrosis Lung Disease 

Rebekka E. Harding-Smith, Deborah R. Gill, Stephen C. Hyde. 

461 

Engineering Mammalian Cells To Secrete RNA to the 

Extracellular Space 

Kevin J. Polach, Leslie Wilkinson, Elaine Brunhoeber, Amy 

Pettigrew, Kirby Wallace, Jeff Sparks, Angela Rea-Ramsey, Casey 

Pence, Majed Matar, Diane McClure, Jennifer Rice, Khursheed 

Anwer, Jason G. Fewell. 

462 

Lipid-Polymer Mediated siRNA Therapy for Silencing 

CXCR4 in Acute Myeloid Leukemia 

Breanne Landry, Hilal Gül-Uludag, Hasan Uludag. 

463 

Messenger RNA as a Novel Therapeutic Approach 

Antonin de Fougerolles. 

464 

Development of Post-Column Process of Immobilized 

Colanic Acid Degrading Enzyme Reactor 

Chromatography in SuperClean Therapeutic Plasmid 

DNA Production 

Li Chen, Christopher M. Jay, Nancy S. Templeton, John Nemunaitis, 

Nicolas Taquet, Phillip B. Maples. 


465 

Cell Replacement Therapy for Parkinson’s Disease with 

Neuronal Cells Derived from Human Parthernogenetic 

Stem Cell 

Ibon Garitaonandia, Rodolfo Gonzalez, Alina Ostrowska, Tatiana 

Abramihina, Gerald Wambua, Alexander Noskov, Francesca 

Boscolo, Louise Laurent, Eugene Redmond, Evan Snyder, Ruslan 

Semechkin. 

466 

Generation of Isogenic Pairs of Mutant and 

Complemented Fanconi Anemia Human Induced 


Laura I. Marquez, Kathryn Schubert, Feorillo Galivo, Milton 

Finegold, Susan Olson, Markus Grompe. 

467 

Engineering of Safety Control Switches for Human 

Induced Pluripotent Stem Cells 

Wu Ou, Pingjuan Li, Jakob Reiser. 

468 

Eficient Generation of Integration-Free iPS Cells from 

Human Adult Peripheral Blood 

Rui-Jun Su, David J. Baylink, Amanda Nieses, Xianmei Meng, Jason 

B. Kiroyan, Kimberly J. Payne, Yuyou Duan, Benjamin Tschudy- 

Seney, Mary Kearns-Jonker, K.-H William Lau, Xiao-Bing Zhang. 

469 

iPSC-Based Strategy To Correct the Bleeding Phenotype 

in Hemophilia A Targeting FVIII Expression to 

Endothelial Cells 

Maria Talmon, Gabriella Ranaldo, Chantal Grosso, Angelo 

Lombardo, Simone Merlin, Stefania E. Cannizzo, Angel Raya, Luigi 

Naldini, Piercarla Schinco, Antonia Follenzi. 


471 

Correction of DF508 CFTR in Patient-Derived iPS Cells 

Using Small Frgement Homologous Replacement 

(SFHR) and Transcription Activator-Like Effector 

Nucleases (TALENs) 

Shingo Suzuki, Thomas J. Cradick, Roy G. Sargent, Lee Albert, 

Matthew Porteus, Gang Bao, Dieter C. Gruenert. 

472 

Directed Differentiation of Human Induced Pluripotent 

Stem (hiPS) Cells into Functional Parathyroid Tissue 

for Cell Therapy 

Alaleh Esmaeili Shandiz, Albert Lee, Shingo Suzuki, R. G. Sargent, 

Michael H. Yezzi, Lisa A. Orloff, Dieter C. Gruenert. 

473 

iPSC-Derived Cardiomyocytes from the Urine of a 

Patient with Duchenne Muscular Dystrophy 

Xuan Guan, David Mack, Yingai Shi, Chad Markert, Guihua Liu, 

Michael Lawlor, Emily Mooreield, Tara Jones, Mark Furth, 

Yuanyuan Zhang, Martin K. Childers. 



121




5:30 PM - 7:30 PM 



474 

Process Development for Scalable Manufacturing of 

hESC-Derived Cardiomyocytes 

Vincent C. Chen, Jingjing Ye, Giau Hua, Ziguang Lin, Dalin Chen, 

Aparna Roy, Patricia Huang, Wei Dang, Josh Tompkins, Arthur 

Riggs, Wenyi Chen, Joseph Wu, David Hsu, Larry Couture. 


476 

Development and Qualiication of Assays To 

Characterize Human Embryonic Stem Cells (hESC) and 

hESC-Derived Products at the Center of Biomedicine 

and Genetics (CBG) 

Patricia Huang, Aparna Krishnan, Wei Dang, Vincent Chen, Jing 

Jing Ye, Aparna Roy, Patricia Lopez, Jing Chai, Ruilin Wu, Tania 

Aguilar, David Hsu, Larry Couture. 



702 

Preclinical study of a scAAV2/8-Lp1-huPPCA vector in 

the treatment of the galactosialidosis mouse model 

Huimin Hu, Elida Gomero, Erik Bonten, John T Gray, Arthur 

Nienhuis, Alessandra d’Azzo 

703 

Improved Retroviral Replicating Vectors for Prodrug- 

Activator Gene Therapy of Cancer 

Noriyuki Kasahara, Christopher Logg, Omar Perez, Oscar Diago, 

Ryan Burnett, Aki Inagaki, Amy Lin, Cindy Burrascano, Mitchel 

Berger, Krystof Bankiewicz, Weijun Wang, Hyun Kim, Walter Wolf, 

Thomas Chen, Kenneth Cornetta, Carlos Ibañez, Joan Robbins, 

Harry Gruber, Douglas J. Jolly 

704 

The recombinant tight junction opener JO-1* decreases 

hypoxia in tumors 

Kamola Saydaminova, Roma Yumul, Christine Wang, Akseli 

Hemminki, Andre Lieber 

705 

Development of a Clinical AAV-Based Vector Encoding 

a Microdystrophin Transgene for the Treatment of 

Duchenne Muscular Dystrophy 

Yoshiaki Miura, Joohee Han, Julia Davydova, Masato Yamamoto 

706 

Novel therapeutic nanoparticles for in vivo delivery of 

low dose siRNA in liver cells and for the treatment of 

liver ibrosis associated nonalcoholic steatohepatitis 

Xavier de Mollerat du Jeu, Akiko Eguchi, Andronikou Nektaria, 

Ariel E. Feldsteinand, Peter Welch 

707 

Directed evolution of artiicial viruses 

Ir. Erik Teunissen, Dr. Enrico Mastrobattista Prof. Dr. Peter Rottier, 

Prof. Dr. Daan Crommelin 

708 

The NanoAssemblr Platform: Microluidics-based 

Manufacture of Limit Size Lipid Nanoparticles for 

Nucleic Acid Delivery 

J. Taylor, C. Walsh, N. Belliveau, P. Lin, R. Rungta, H. Choi, J. Lee, E. 

Ramsay, T. Leaver, A. Wild, K. Ou, A. Leung, Y. Tam, I. Hafez, S. Chen, 

B. MacVicar, C. Hansen, P. Cullis 

709 

Preclinical development of an AAV vector for the 

treatment of X-linked retinitis pigmentosa due to 

RPGRorf15 mutation 

Zhijian Wu, Suja Hiriyanna, Haohua Qian, Suddhasil Mookherjee, 

Kayleigh Kaneshiro, Maria Campos, Chun Gao, Robert Fariss, 

Anand Swaroop, Tiansen Li, Peter Colosi 

710 

Cytotoxic T-cell Surface Engineering with Chemically 

Self-Assembled Antibody Nanorings (CSANS) 

C. R. Wagner, Kari Gabrielse, Dr. Jae Chul Lee 

711 

Speciic siRNA delivery to primary activated T cells for 

anti-inlammatory therapy 

Na Hyung Kim, Yuran Xie, Archana Thakur, Lawrence Lum, Olivia 

Merkel 

712 

MicroRNA-155 confers encephalogenic potential to 

Th17 cells by promoting effector gene expression 

Ruozhen Hu, Thomas B. Huffaker, Dominique A. Kagele, Marah C. 

Runtsch, Erin Bake, Aadel A. Chaudhuri, June L. Round, Ryan M. 

O’Connell 

713 

DNA Mini Strings: The Gold Standard for Transgene 

Delivery 

Naiseh Naissi, Roderick Slavcev 

714 

Restoring a minimal motor unit potential and 

peripheral motor conductivity in lower extremities in 

a 15 years old girl with complete loss of spinal cord 

continuity at Th2-Th3 after combine autologous bone 

marrow derived cells and mesenchymal stem cells tr 

Jarocha Danuta, Milczarek Olga, Kwiatkowski Stanislaw, Majka 

Marcin 

715 

Genomic correction of Duchenne Muscular Dystrophy 

patient-derived iPSCs using TALENs 

Hongmei Li, Naoko Fujimoto, Noriko Sasakawa, Takashi 

Yamamoto, Knut Woltjen, Hidetoshi Sakurai, Shinya Yamanaka, 

Akitsu Hotta 

122 




5:30 PM - 7:30 PM 



716 

A versatile method for titration of chimeric antigen 

receptor-expressing retroviral vectors based on 

translocation of CD3-epsilon 

Daniel Abate-Daga, Steve A. Feldman, Rachel E. Beard, Steven A. 

Rosenberg, Richard A. Morgan 

717 

Dual Speciic Suicide Gene Expression plasmid Delivery 

Using Bio-reducible Polymer for Hepatocellular 

Carcinoma Gene Therapy 

Hyun Ah Kim, Kihoon Nam, Minhyung Lee, Sung Wan Kim 

723 

Attenuation of TGF-β induced liver ibrosis by bone 

morphogenetic protein 2. 

Mu-Rou Tsai, Pao-Rong Chen, Yu-Lin Yang,, Ming-Chuan Hung, 

Tao-Chen Lee, Tsuing-Jeu Hung, Chien-Ya Hung, Wen-Teng Chang, 

Pei-fang Hsieh Shu-Fen Liu 

724 

Subpopulation of T-lymphocytes in leukoconcentrates 

obtained from bone marrow donors in the setting of 

stable hematopoiesis 

R. Bogdanov, L. Mendeleeva, I. Galtseva, L. Kuzmina, T. Gaponova, 

N. Kalinin, E. Parovichnikova, V. Savchenko 

718 

Expression of the immunomodulatory cytokines 

interleukin-10 and interleukin-6 are increased in 

equine MSCs cultured in an inlammatory environment 

in vitro 

KO Ortved, J Witten, AJ Nixon 

719 

Gene Delivery Using Therapeutic Ultrasound To 

Mesenchymal Stem Cells: Targeted Antiangiogenic 

Tumor Therapy 

Tom Haber, Marcelle Machluf 

720 

The role of S100A4 in glioblastoma and cancer stem 

cell 

Shih-Hwa Chiou, Jun-Ming Han, Hsin-I Ma 

721 

Biodistribution and toxicology studies of AAV2/rh8- 

CBA-β-hexosaminidase after intracranial delivery in 

C57/Bl6 mice 

Kirsten Erger, Ronald Mandel, William Castleman, Martha 

Campbell-Thompson, Corinne Abernathy, Matthew Getz, Travis 

Cossette, Lynda Schneider, Ryan Brown, Miguel Sena-Esteves, 

Thomas Conlon 


722 

Qualiication for human umbilical cord derived 

mesenchymal stem cells (hUC-MSCs) for regenerative 

medicine 

Hyun Woo Lee, Su-Youne Han, Hyeon Kyeong Lee, Seung Hoon Pak, 

Jin Tac Kim, Ji Hyang Kim, Byung-Rok Do 


123

Abstract Directory, Saturday, May 18, 2013 


Late Breaking Abstracts 

10:15 am - 12:15 pm 




10:15 AM - 12:15 PM 

Room: 150 ABC 


725 10:15 AM 

AAV/RNAi-mediated improvement of anti-malaria 

vaccination strategies 

Christiane Hammerschmidt-Kamper, Franziska Hentzschel, 

Kirsten Heiss, Kathleen Boerner, Ann-Kristin Mueller, Dirk Grimm 

726 10:30 AM 

Post-entry Restriction of AAV Vectors by A PHD Finger 

Protein 

Claire A. Shreiber, Toshie Sakuma, Yasuhiro Ikeda 

727 10:45 AM 

AAV-mediated Bevacizumab Maintenance Therapy 

Inhibits Ovarian Cancer In Vivo 

Yi Xie, PhD, Martin J. Hicks, PhD, Stephen M. Kaminsky, PhD, 

Malcolm A.S. Moore, PhD, Ronald G. Crystal, MD, Arash Raii, MD, 

PhD 

728 11:00 AM 

Human iPS-derived antigen-targeted T cells are 

functional innate-like cytotoxic effectors and lyse 

CD19-positive tumor cells 

Maria Themeli, Christopher C. Kloss, Giovanni Ciriello, Michel 

Sadelain 

729 11:15 AM 

Penton-dodecahedral particles trigger opening of 

intercellular junctions and facilitate viral spread 

during adenovirus serotype 3 infection of epithelial 

cells. 

Zhuo-Zhuang Lu, Hongjie Wang, YiYi Zhang, Zongyi Li, Pascal 

Fender, Andre Lieber 

730 11:30 AM 

An oncolytic adenovirus able to express a full-length 

functional anti-Her2 monoclonal antibody from tumor 

cells 

Paula Savola, Ilkka Liikanen, Theresia Gutmann, Kilian Guse and 

Akseli Hemminki 

731 11:45 AM 

Non-toxic and highly eficient targeting of a human 

chromosomal locus with a lentiviral vector-associated 

meganuclease 

Chenxia He, Agnès Gouble, Alix Bourdel, Laurent Poirot, Frédéric 

Pâques, Philippe Duchateau, Aleksander Edelman, Olivier Danos 

732 12:00 PM 

Non-viral gene delivery to human embryonic stem cellderived 

retinal pigment epithelium 

A Subrizi, T Ilmarinen, M Yliperttula, H Tenhu, H Skottman, A Urtti 

477 10:15 AM 

Molecular Modeling of Coagulation FVII Binding to 

Adenovirus Hexon Revealed a Binding Pocket for 

Coagulation Factor GLA Domain That Deines the 

Infectivity of Adenovirus-Coagulation Factor Complexes 

Eric E. Irons, Justin W. Flatt, Konstantin Doronin, Tara L. Fox, 

Mauro Acchione, Phoebe L. Stewart, Dmitry M. Shayakhmetov. 

478 10:30 AM 

Identiication of a Negative Regulator of TRPV1 Via an 

HSV Based cDNA Library Screen 

Bonnie L. Reinhart, Asaff Harel, MingDi Zhang, James R. Goss, 

William F. Goins, Justus B. Cohen, Joseph C. Glorioso. 

479 10:45 AM 

Long Term Follow Up of Phase II Clinical Trial of a 

Granulocyte-Macrophage Colony-Stimulating Factor- 

Encoding, Second-Generation Oncolytic Herpes Virus 

(Talimogene Laherparepvec, T-VEC) in Patients with 

Unresectable Metastatic Melanoma 

John Nemunaitis, Cynthia Bedell, Beena O. Pappen, Staci Horvath, 

Neil Senzer. 

480 11:00 AM 

Combined Therapies with Oncolytic Adenovirus 

Suppress Tumor Growth in Hamsters 

Brittany A. Young, Karoly Toth, Jacqueline F. Spencer, William S. M. 

Wold. 

481 11:15 AM 

Analysis of Host Cell and Viral Transcriptomes during 

Infection by Wild-Type Species C Ad6 and Species D 

Ad26 

Mallory A. Turner, Sean E. Hoherr, Michael A. Barry. 

482 11:30 AM 

Suppression of the Leaky Expression of Adenovirus 

Genes Following Transduction with a Replication- 

Incompetent Adenovirus Vector by Incorporating 

microRNA-Targeted Sequences into the 3’-Untranslated 

Region of the E2A, E4, or pIX Gene 

Kahori Shimizu, Fuminori Sakurai, Shin-ichiro Nakamura, Yasuhito 

Nagamoto, Kyoko Tomita, Masashi Tachibana, Hiroyuki Mizuguchi. 

483 11:45 AM 

Biodistribution of Fiber-Chimeric Ad5/3 Vectors in 

DSG2-Transgenic Mice 

Maximillian Richter, Roma Yumul, Hongjie Wang, Dirk Nettelbeck, 

Andre Lieber. 

124 





10:15 AM - 12:15 PM 

Room: 150 ABC 

484 12:00 PM 

Ad5-Based Vectors Can Transduce Hepatocytes with 

High Eficiency Independent of FX 

Jan-Michael Prill, Lea Krutzke, Tatjana Engler, Stefan Kochanek, 

Florian Kreppel. 


Nuclease Mediated Genome Editing 

10:15 AM - 12:15 PM 


485 10:15 AM 

Cross Your Fingers: A Comprehensive Survey of the 

Cys2His2 Zinc Finger Domain Hints toward Codes of 

Speciicity and Assembly 

Elizabeth F. Rowland, Anton V. Persikov, Benjamin L. Oakes, Mona 

Singh, Marcus B. Noyes. 

486 10:30 AM 

Eficient Site-Speciic Integration and In Situ Gene 

Correction of Human Long-Term Repopulating 

Hematopoietic Stem Cells by Zinc Finger Nucleases 

Pietro Genovese, Angelo Lombardo, Giulia Schiroli, Giulia Escobar, 

Tiziano Di Tomaso, Claudia Firrito, Davide Moi, Lucia Sergi Sergi, 

Philip D. Gregory, Michael C. Holmes, Luigi Naldini. 

487 10:45 AM 

Optimizing Plasmid DNA Templates for Homologous 

Recombination-Mediated Gene Modiication Following 

Zinc Finger Nuclease Treatment of Hematopoietic Stem 


Colin M. Exline, Orla L. Mulhern, Nick Llewellyn, Jill Henley, Jianbin 

Wang, Kenneth Kim, Jenny Yan, Philip D. Gregory, Michael C. 

Holmes, Paula M. Cannon. 

488 11:00 AM 

In Vivo ZFN Mediated Targeting of Albumin as a 

Platform for Expression of Multiple Therapeutic Genes 

Rajiv Sharma, Xavier M. Anguela, Yannick Doyon, Sunnie Y. Wong, 

David E. Paschon, Hojun Li, Virginia Haurigot, Robert J. Davidson, 

Shangzhen Zhou, Philip D. Gregory, Michael C. Holmes, Edward J. 

Rebar, Katherine A. High. 

489 11:15 AM 

Quantifying Rates of Gene Targeting and Off-Target 

Cleavage of Engineered Nucleases Using SMRT 

Sequencing 

Eli J. Fine, Eric Kildebeck, Yanni Lin, Matthew H. Porteus, Thomas 

J. Cradick, Gang Bao. 

490 11:30 AM 

Precise Modiication of Human Genes Directed by 

Single-Stranded DNA Oligonucleotides and TALENs 

Shengdar Q. Tsai, Deepak Reyon, Cyd Khayter, J. Keith Joung. 

491 11:45 AM 

Eficient Template-Free Genetic Correction with TALE 

Nucleases 

David G. Ousterout, Pablo Perez-Pinera, Pratiksha I. Thakore, 

Ami M. Kabadi, Matthew T. Brown, Kamel Mamchaoui, Jacques P. 

Tremblay, Charles A. Gersbach. 

492 12:00 PM 

Enhancing the Eficiency of Zinc Finger Nuclease 

Delivery by Integrase-Defective Lentiviral Vectors Using 

Valproic Acid 

Alok V. Joglekar, Michelle Ho, Libby Stein, Sara Sanadiki, Roger P. 

Hollis, Donald B. Kohn. 



and Genetic Diseases II 

10:15 AM - 12:15 PM 


494 10:15 AM 

Hematopoietic Stem Cell Gene Therapy for Cystinosis 

Sarah Ur, Celine J. Rocca, Jay Sharma, Donald B. Kohn, Stephanie 

Cherqui. 

493 10:30 AM 

Mechanism of Hematopoietic Stem Cell-Mediated 

Therapy in Cystinosis 

Brian A. Yeagy, Jay Sharma, Celine J. Rocca, Sarah Ur, Thomas 

Whisenant, Daniel R. Salomon, Stephanie Cherqui. 

495 10:45 AM 

Prevention of Neurocognitive Deicit by Ex Vivo 

Lentiviral Transduction of Hematopoietic Stem Cells in 

a Murine Model of Mucopolysaccharidosis Type II 

Kelly M. Podetz-Pedersen, Andrea D. Karlen, Megan P. Buckvold, 

Zenhong Nan, Vladimir Slepushkin, Andre Roy, Galina Yakovief, 

Zi Gong, Joseph Muenzer, Boro Dropulic, Walter C. Low, R. Scott 

McIvor. 

496 11:00 AM 

Hyper-Functional Synthetic Transgenes Improve the 

Eficiency of Lentiviral and AAV-Based Liver-Directed 

Gene Therapy and Induce Immune Tolerance 

Nisha Nair, Alessio Cantore, Mario Di Matteo, Patrizia Della Valle, 

Jessica Willems, Hanneke Evens, Armando D’Angelo, Janka Matrai, 

Luigi Naldini, Marinee Chuah, Thierry VandenDriessche. 



125






10:15 AM - 12:15 PM 


497 11:15 AM 

Prevention of Neuropathology and Normal Cognitive 

Development in the Hyperargininemic Mouse with AAV 

Gene Transfer 

Ellen K. Lee, Chuhong Hu, Ragini Bhargava, Ravi Ponnusamy, Hana 

Park, Sarah Novicoff, Nora Rozengurt, Bart Marescau, Peter De 

Deyn, David Stout, Lisa Schlichting, Wayne W. Grody, Stephen D. 

Cederbaum, Gerald S. Lipshutz. 

498 11:30 AM 

Assessing the Safety of AAV Liver Gene Transfer of 

Hyperfunctional FIX Padua in Inhibitor-Prone Dogs and 

Mice 

Julie M. Crudele, Jonathan D. Finn, Joshua I. Siner, Yifeng Chen, 

Shangzhen Zhou, Glenn P. Niemeyer, Clinton D. Lothrop Jr, Federico 

Mingozzi, Katherine A. High, Valder R. Arruda. 

499 11:45 AM 

Development of AAV2/8-Mediated Gene Therapy 

Clinical Trial for Crigler-Najjar Syndrome Type I: 

Optimization of Liver-Speciic Expression Cassette 

Nunzia Pastore, Alberto Auricchio, Nicola Brunetti-Pierri. 

500 12:00 PM 

Stromal Cell-Derived Factor-1 Non-Viral DNA Therapy 

Accelerates Wound Healing and Decreases Scar 

Formation in Porcine Surgical Incisions 

Timothy J. Miller, Karissa Henson, Rahul Aras, Evan Facher, Marc 

S. Penn. 


Cancer Immunotherapy II 

10:15 AM - 12:15 PM 


501 10:15 AM 

DAI-Armed Double Deleted Oncolytic Vaccinia Virus 

Displays Enhanced Anti-Tumor Activity by Eliciting a 

More Robust Anti-Tumor Immune Response 

Mari Hirvinen, Kilian Guse, Marko Ahonen, Eerika Karli, Dario 

Greco, Markus Vähä-Koskela, Akseli Hemminki, Vincenzo Cerullo. 

502 10:30 AM 

Clinical Implications of ROR1-Speciic T Cells That 

Target B-Cell Leukemia 

Drew C. Deniger, Thomas H. Kipps, Simon Olivares, Harjeet Singh, 

Sourindra Maiti, Lenka V. Hurton, Kirsten C. Switzer, Tiejuan Mi, 

Radhika Thokala, Helen Huls, William G. Wierda, Laurence J. N. 

Cooper. 

503 10:45 AM 

Randomized Phase II Trial of Adjuvant Autologous 

Tumor Cell Vaccine (FANG) for High Risk Stage III/IV 

Ovarian Cancer: Preliminary Results 

Neil Senzer, Minal Barve, Cynthia Bedell, Jonathan Oh, Joseph 

Kuhn, Bruce Fine, Thomas P. Heffernan, Carolyn M. Matthews, C. 

Allen Stringer, E. Colin Koon, Samuel Lifshitz, Beena O. Pappen, 

Padmasini Kumar, Phillip B. Maples, John Nemunaitis. 

504 11:00 AM 

TCR Gene Editing To Treat Acute Leukemia and 

Multiple Myeloma 

Sara Mastaglio, Pietro Genovese, Zulma Magnani, Elena Provasi, 

Angelo Lombardo, Andreas Reik, Nicoletta Cieri, Aurora Brambilla, 

Fabio Ciceri, Claudio Bordignon, Michael C. Holmes, Philip D. 

Gregory, Luigi Naldini, Chiara Bonini. 

505 11:15 AM 

Novel VSV-Based Immunotherapy for Brain Tumors 

Rosa Diaz, Diana Rommelfanger-Konkol, Timothy Kottke, Jill 

Thompson, Oliver Donnelly, Addie Embry, Alan Melcher, Richard 

Vile. 

506 11:30 AM 

CD19-Targeted T Cells Rapidly Induce Molecular 

Remissions in Adults with Chemotherapy Refractory 

Acute Leukemia 

Renier Brentjens, Marco L. Davila, Isabelle Riviere, Jae Park, 

Xiuyan Wang, Shirley Bartido, Jolanta Stefankski, Oriana Borquez- 

Ojeda, Yvette Bernal, Michel Sadelain. 

507 11:45 AM 

A Vaccinia Virus Armed with Interleukin-10 That 

Dampens Host Immunity to the Vctor but Not to Tumor 

Is a Promising Therapeutic Agent for Pancreatic Cancer 

Louisa S. Chard, Eleni Maniati, Pengju Wang, Jonathan Hughes, 

Jiwei Wang, Bela Denes, Istvan Fodor, Thorsten Hagemann, 

Nicholas R. Lemoine, Yaohe Wang. 

508 12:00 PM 

Identiication of an Alternative Speciicity for 

Engineered T Cells Expressing an Enhanced Afinity 

MAGE A3 TCR 

Namir J. Hassan, Brian Cameron, Andrew Gerry, Joseph Dukes, 

Jane Harper, Vivekanandan Kannan, Frayne Bianchi, Francis 

Grand, Giovanna Bossi, Joanna Brewer, Debbie Sutton, Annelise 

Vuidepot, Alex Powlesland, Alison Legg, Katherine Adams, Alan 

Bennett, Nick Pumphrey, Gwen Binder-Scholl, Irina Kulikovskaya, 

Minnal Gupta, Gabriela Plesa, Michael Kalos, Bruce L. Levine, 

James L. Riley, Angel Varela-Rohena, Edward A. Stadtmauer, Aaron 

P. Rapoport, Gerald P. Linette, Carl H. June, Bent Jakobsen. 

126 





10:15 AM - 12:15 PM 


509 10:15 AM 

Targeted, Multifunctional RNA Therapies for Advanced 

Cancers of the Prostate 

Luiza I. Hernandez, William M. Rockey, Craig A. Howell, Justin P. 

Dassie, Frank J. Hernandez, Xiu Y. Liu, Paloma H. Giangrande. 

510 10:30 AM 

Circulating Plasmid Detection in a Phase I Clinical Trial 

of Intratumoral Injection of pbi-shRNA Nanoplex 

Christopher M. Jay, Donald D. Rao, Zhaohui Wang, Neil Senzer, 

Phillip B. Maples, John Nemunaitis. 

511 10:45 AM 

Smooth Muscle Cell Targeted RNA Aptamers for the 

Treatment of Vascular Disease 

William H. Thiel, Maysam Takapoo, Shuxia Jiang, Jennifer 

Streeter, Bojana Stanic, Xiuying Liu, Francis J. Miller, Jr, Paloma H. 

Giangrande. 

512 11:00 AM 

Long-Term Inhibition of miR-122 Signiicantly 

Increases Hepatocellular Carcinoma in Multiple Adult 

Mouse Models 

Jun Xie, Qin Mao, Jia Li, Xiaofang Yang, Qin Su, Ran He, Zhong Jiang, 

Xiaofei Wang, Phillip D. Zamore, Guangping Gao. 

513 11:15 AM 

In Vitro and In Vivo Effects of Composite Calcium 

Phosphate and Poly(Lactic-Co-Glycolic Acid) (PLGA) 

Nanoparticles Engineered for Enhanced Cancer Cell 

Internalization and Sustained siRNA Release 

Lisa A. Tobin, Yili Xie, Su I. Chung, King F. Kwong. 

514 11:30 AM 

Robust RNAi Enhancement Via Human Argonaute-2 

Overexpression from Plasmids, Viral Vectors or Cell 

Lines 

Kathleen Boerner, Dominik Niopek, Daniel Gilbert, Konrad Streetz, 

Hans-Georg Kraeusslich, Dirk Grimm. 

515 11:45 AM 

Ultra-Stable Trivalent RNA Nanoparticles for Targeting 

to Cancers and HIV-1 

Kato Shum, Peixuan Guo, John J. Rossi. 

516 12:00 PM 

Directed Evolution-Based Dissection of the Four Human 

Argonaute Proteins 

Nina Schuermann, Leonardo Trabucco, Christian Bender, Rob 

Russell, Dirk Grimm. 

Poster Session III 

3:45 PM - 5:45 PM 



517 

Data Mining Methods for Integration Site Analysis in 

Hematopoietic Stem Cell Gene Therapy Trials 

Andrea Calabria, Luca Biasco, Fabrizio Benedicenti, Simone Leo, 

Martina Cesani, Gianluigi Zanetti, Elia Stupka, Alessandra Bifi, 

Alessandro Aiuti, Luigi Naldini, Eugenio Montini. 

518 

Mutually Dependent Barcode Strategy for Accurate 

Deep Sequencing of Vector Integration Sites 

Richard Gabriel, Cynthia C. Bartholomae, Frank Giordano, Raffaele 

Fronza, Stephanie Laufs, Christof von Kalle, Manfred Schmidt. 

519 

Sequencing of a Genetic Barcode Reveals Altered 

Population Dynamics in a Mouse Model of Transplanted 

Hematopoietic Stem Cells Transduced with a Lentivirus 

Encoding LMO2 

Bryan E. Strauss, Daniela B. Zanatta, Rodrigo Aguiar, Maristela 

Tsujita, Primavera Borelli. 

520 

A New Strategy for Retroviral Vector Puriication Based 

on LNGR-Ab Immunomagnetic Selection 

Stornaiuolo Anna, Corna Stefano, Bossi Sergio, Bordignon Claudio, 

Rizzardi Gian Paolo, Bovolenta Chiara. 

521 

Lentiviral Gene Therapy in a SCID-X1 Mouse Model 

after Treatment with Granulocyte Colony Stimulating 

Factor 

Marshall W. Huston, Niek P. van Til, Rana Yadak, Yvette van 

Helsdingen, Gerard Wagemaker. 

522 

Development of Human Mesenchymal Stem Cell-Based 

Tumor-Homing Producer Cells for Eficient Delivery of 

Retroviral Replicating Vectors to Brain Tumors 

Shuichi Kamijima, Akihito Inagaki, Kei Hiraoka, Masamichi 

Takahashi, Quincy Tam, Emmanuelle Faure-Kumar, Janet Treger, 

Brooke Bogan, Christopher R. Logg, Noriyuki Kasahara. 

523 

Reducing In Vivo Clearance of Lentiviral Vectors by 

Immune Cells in order To Improve Eficacy and Safety 

of Gene Therapy 

Nisha G. Sosale, Irena I. Ivanovska, Richard K. Tsai, Philip K. 

Zoltick, Yale Goldman, Dennis E. Discher. 

524 

A Novel Retroviral Envelope as a Candidate for 

Pseudotyping Retroviral and Lentiviral Vectors 

Wenqin Xu, Jill Russ, Kristen Gorman, Kyle Delaney, Maribeth V. 

Eiden. 



127


Poster Session III, continued 

3:45 PM - 5:45 PM 



525 

A Novel Shuttle Vector Approach To Identify Genes 

Involved in Hematopoietic Stem Cell Disorders Using 

Vector-Mediated Genotoxicity 

Dustin T. Rae, Ryan Maynard, H. Joachim Deeg, Grant D. Trobridge. 

526 

A Systematic Approach to the Establishment and 

Characterisation of Endothelial Progenitor Cells for 


Yuan Zhao, Natalie Werling, Robin Thorpe. 

527 

Lentiviral Vector Transduction of Human CD34+ Cells 

Requires a Wash/Rest Phase between Vector Exposures 

To Produce Additional Beneit but Does Not Require 

High Dose Cytokines 

Michael R. Greene, Brian P. Sorrentino. 

528 

Phorbol 12-Myristate 13-Acetate Enhances Lentiviral 

Transduction of Human Hematopoietic Cells through 

Activation PKC 

Jennifer M. Johnston, Gabriela Denning, Christopher B. Doering, H. 

Trent Spencer. 

529 

Production of Recombinant Lentiviral Vectors 

in Serum-Free Suspension Culture and Column 

Chromatography Puriication 

Yonghai Li, Bernd Hauck, Tiffany Kincaid, Fraser J. Wright, 

Katherine A. High, Guang Qu. 

533 

Lentiviral Vectors-Mediated Long-Term and High 

Eficiency Expression of Foreign Gene in HEK 293T Cells 

Renhe Yan, Yingying Mao, Zhibing Liang, Yi Feng, Tianbai Li, 

Maomin Lu, Weiwang Gu, Jingang Zhang, Hongwei Li. 

534 

Tumorigenic Effect of Transplanted Myoblasts 

Overexpressing Bcl-2 Based on Retroviral Vector 

Francisco Martinez-F, Maria Ustoa, Araceli Barrera-L, Hugo 

E. Sandoval-Zamora, Bruno Guevara, Pablo Vizcaino-Dorado, 

Alejandro Zentella-Dehesa, Catalina Machuca-R. 

535 

Transduction of Ferret Airways with Avian Inluenza 

Virus Hemagglutinin Pseudotyped Equine Infectious 

Anemia Virus Vector 

Ziying Yan, Manij Patel, Xingshen Sun, Diana CM - Lei-butters, 

Hongshu Sui, John C. Olsen, John F. Engelhardt. 

AAV Vectors III 

536 

Multiple Molecular Alterations in Phosphodegrons 1-3 

within AAV2 Capsid Demonstrates Higher Hepatic Gene 

Transfer Eficiency 

Dwaipayan Sen, V. Kalaivani, Rupali Gadkari, G. Sudha, N. 

Srinivasan, Alok Srivastava, Giridhara R Jayandharan. 

537 

Improved Adeno-Associated Virus Serotype (AAV)5 

Vectors for Hepatic Gene Therapy 

Dwaipayan Sen, Prachi Agarwal, V. Kalaivani, Alok Srivastava, 

Giridhara R Jayandharan. 

538 

Adeno Associated Virus Puriication Independent of 

Gradient Centrifugation and Chromatography 

Erik Arden, Joseph M. Metzger. 


530 

Eficient Selection of Genetically Engineered HIV 

Resistant Cells by Short Hairpin RNA Mediated HPRT 

and CCR5 Knockdown 

Munetoshi Narukawa, Saki Shimizu, Katrin Hacke, Janet Treger, 

Ruth Cortado, Erica Egger, Noriyuki Kasahara, Dong Sung An. 

531 

Designing Trans-Regulated Lentiviral Vectors for Gene 

Transfer in Functional Genomics and Gene Therapy 

Agnes Holczbauer, Adriana Zingone, Suresh Arya. 

532 

Application of VSV-G Induced Nanovesicles (Gesicles) 

for the Delivery of Active Protein To Target Cells 

Thomas P. Quinn, Lily Lee, Mei Fong, Michael Haugwitz, Hiroaki 

Sagawa, Andrew Farmer. 

539 

Transcriptional Repression with Zinc-Finger and Tale 

Protein Scaffold 

Salvatore Botta, Elena Marrocco, Nicola de Prisco, Toni Cathomen, 

Claudio Mussolino, Enrico Maria Surace. 

540 

Mouse Gender Inluences Brain Expression of a 

Reporter Gene by Intravascularly-Administered AAV9 

Casey A. Maguire, Matheus H. W. Crommentuijn, Dakai Mu, Eloise 

Hudry, Alberto Serrano-Pozo, Bradley Hyman, Bakhos A. Tannous. 

541 

Site-Directed Mutagenesis of Surface-Exposed 

Lysine Residues Leads to Improved Transduction 

by Recombinant AAV2 and AAV8 Vectors in Murine 

Hepatocytes In Vivo 

Baozheng Li, Wenqin Ma, George Aslanidi, Chen Ling, Kim Van 

Vliet, Mavis Agbandje-McKenna, Arun Srivastava. 

128 




3:45 PM - 5:45 PM 


AAV Vectors III 

542 

Transduction of Murine, Nonhuman Primate, 

and Human Hematopoietic Stem Cells (HSCs) by 

Recombinant AAV Serotype Vectors: Identiication of 

AAV6 as the Most Eficient Serotype for Human HSCs, 

and Further Augmentation in Transduction Eficiency 

In Vitro and In Vivo with Point-Mutations of Tyrosine 

Residues in the Viral Capsid 

Liujiang Song, M. Ariel Kauss, Etana Kopin, Manasa Chandra, 

Tahira Ul-Hasan, Erin Miller, Giridhara R. Jayandharan, Angela 

E. Rivers, George V. Aslanidi, Chen Ling, Baozheng Li, Wenqin 

Ma, Xiaomiao Li, Lourdes M. Andino, Li Zhong, Alice F. Tarantal, 

Mervin C. Yoder, Kamehameha K. Wong, Jr, Menqun Tan, Saswati 

Chatterjee, Arun Srivastava. 

543 

Eficient Transduction of Canine Muscle Using rAAV6 

Jane T. Seto, Julian N. Ramos, James M. Allen, Eric Finn, Guy L. 

Odom, Jeffrey S. Chamberlain. 

544 

Integrated Transcriptional and Post Transcriptional 

Targeting Results in Cardiac Biased Transgene 

Expression 

Jinhui Wang, Joshua Gorsky, Jonathan Peterson, Joseph 

Rabinowitz. 

545 

Reprogramming Adipose Tissue Derived Mesenchymal 

Stem Cells (AT-MSC) into Pluripotent Stem Cells by a 

Mutant AAV Vector 

Mong-Jen Chen, Yuanqing Lu, Takashi Hamazaki, Hsin-Yin Tsai, 

Kirsten Erger, Thomas Conlon, Arun Srivastava, Mark Brantly, 

Vince Chiodo, William Huaswirth, Naohiro Terada, Sihong Song. 

546 

Optimized Kidney-Targeted Gene Delivery Using AAV in 

a Mouse Model of Cystinosis 

Celine J. Rocca, Sarah M. Ur, Corinne Antignac, Jude R. Samulski, 

Stephanie Cherqui. 

547 

Concentration of AAV8 and 9 Vector to Titers Ranging 

from 1-6 x1014 vg/mL: Feasibility Assessment for 

Volume-Limited Dosing 

J. Fraser Wright, Bernd Hauck, Shang-Zhen Zhou, Olga Zelenaia, 

Marina Sumaroka, Katherine A. High. 

548 

Enhanced Fracture Healing Utilizing Directed Evolution 

of Adeno-Associated Virus 

Seth G. Levy, Damien Marsic, Shawn Gilbert, Sergei Zolotukhin, 

Selvarangan Ponnazhagan. 

549 

Targeted Bio-Engineering of Adeno Associated Virus 

Serotype rh.10 Capsid Improves Its Gene Delivery In 

Vitro and In Vivo 

Ruchita Selot, Nishanth Gabriel, Monika Kumari, Giridhara R 

Jayandharan. 

550 

Adeno-Associated Virus Vectors Containing Either 

Elongation Factor 1 alpha or Human Synapsin 1 

Promoters Eficiently Drive Expression in Rodent 

Primary Cortical Neurons 

Thomas G. Wolfe, Yun Lin, Holly Carlisle, Danny Ortuno, Huiren 

Zhao, Yun Hu, Yu Sun, Cherylene Plewa, Paul Kassner, Ki Jeong Lee. 

551 

Highly Flexible, Precise and Eficient Gene Editing of 

Diverse Human Tissue Types Using rAAV-Mediated 

Homologous Recombination 

Ramu Mangena, Tom Henley, Christine Schoield, Chris Torrance, 

Eric Rhodes. 


552 

Structural Studies on the Interaction of Adenovirus 

Serotype 3 Fiber Knob and Desmoglein 2 

Hongjie Wang, Roma Yumul, Maximillian Richter, Pascal Fender, 

Andre Lieber. 

553 

Blocking of Scavenger-Receptor A Results in Increased 

HDAd-Mediated Hepatocyte Transduction through 

Inhibition of Vector Particle Uptake by Kupffer Cells 

and Liver Sinusoidal Endothelial Cells 

Pasquale Piccolo, Pratibha Mithbaokar, Nicola Brunetti-Pierri. 

554 

Artiicial Riboswitches for External Regulation of 

Oncolytic Viruses 

Patrick Ketzer, Johanna K. Kaufmann, Sarah Engelhardt, Sascha 

Bossow, Jörg S. Hartig, Guy Ungerechts, Dirk M. Nettelbeck. 

555 

Liver-Detargeted Ad5/48 Chimaeric Hexon Based 

Oncolytic Adenoviruses Targeting TGFb Signaling: 

A Safe and Effective Approach for the Treatment of 

Prostate Cancer Bone Metastases 

Weidong Xu, Zhenwei Zhang, Zebin Hu, Stuart Stock, Charles 

Brendler, Prem Seth. 



129




3:45 PM - 5:45 PM 



556 

Enhanced Anti-Tumor Eficacy by Oncolytic Adenovirus 

Using a Stimuli Responsive Block Copolymer 

mPEG-b-Poly(His) 

Joung-Woo Choi, You Han Bae, Chae-Ok Yun. 

557 

Distinct Integration Proiles after Sleeping Beauty 

Transposase-Mediated Somatic Integration from AAV 

and Adenoviral Vectors 

Wenli Zhang, Christina Rauschhuber, Martin Haeusl, Nadine 

Muether, Anja Ehrhardt. 

558 

Screening and Characterization of Alternative 

Adenovirus Serotypes with Enhanced Virus- 

Lateralization Ability In Vitro for Improved Antitumor 

Eficacy 

Junji Uchino, David T. Curiel, Hideyo Ugai. 

559 

Improved Animal Models for Testing Gene or Cell 

Therapies for Atherosclerosis 

Liang Du, Jingwan Zhang, David Dichek. 

560 

Liver Retargeting of Adenovirus Using Robo4 Promoter 

and Hexon Substitution 

Sergey A. Kaliberov, Lyudmila N. Kaliberova, Zhi Hong Lu, 

Meredith A. Preuss, Justin A. Barnes, Cecil R. Stockard, William E. 

Grizzle, Jeffrey M. Arbeit, David T. Curiel. 

561 (Moved to Poster Session I after Abstract 138) 

Lister Strain Vaccinia Virus Demonstrates Favourable 

Anti-Tumor Eficacy and Tumor-Selectivity Compared 

to Western Reserve Strain 

Jonathan Hughes, Pengju Wang, Ghassan Alusi, Vipul Bhakta, 

Yongchao Chu, Hailiang Shi, Iain McNeish, Andrea McCart, Istvan 

Fodor, Nicholas Lemoine, Yaohe Wang. 

562 

Development of a Novel Vaccine Vector Based on 

Synthesized Adenovirus Genome 

Yong Yang, Chao Zhang, Xiang Wang, Yudan Chi, Dongming Zhou. 

563 

Construction of a Novel Bicistronic Adenoviral Vector 

Feilong Jie, Shengyao Wang, Jie Luo, Na Zhang, Jingang Zhang, 

Hongwei Li. 

564 

Ciproloxacin Regulates Negatively Egr-1 

Transcriptional Activity in Human Primary Tenocytes 

Transduced with Ad-Egr-1/Luc 

Francisco Martinez-F, Araceli Barrera-Lopez, Hugo Sandoval- 

Zamora, Karina Guzman-M, Alejandro Jimenez-Orozco, Christian Y. 

Lomeli-R, David T. Curiel, Rebecca E. Franco-Bourland. 


565 

A Placebo Controlled Phase IIb Clinical Studyof 

TissueGene-C (TG-C) in Patients with Osteoarthritis 

Jung Jong Cho, Tae Won Kim, Yeo Myeong Park, Eu Gene Jeong, 

Moon Jong Noh, Kwan Hee Lee, Bum Sup Lee. 

566 

A Novel Oncolytic HSV Design Based on the Common 

Over-Expression of miR-21 in Tumors 

Marco Marzulli, Lucia Mazzacurati, Bonnie Reinhart, Mark E. 

Hatley, Joseph C. Glorioso, Paola Grandi, Justus B. Cohen. 

567 

Comparing the Activity of TALENs Constructed with 

Different Guanine-Targeting RVDs 

Yanni Lin, Thomas J. Cradick, Eli J. Fine, Gang Bao. 

568 

In Vivo Cleavage of Transgene Donors Promotes 

Nuclease-Mediated Targeted Integration 

Sandra Cristea, Yevgeniy Freyvert, Yolanda Santiago, Michael C. 

Holmes, Fyodor D. Urnov, Philip D. Gregory, Gregory J. Cost. 

569 

Helper Independent Self-Inactivating Chimeric 

PiggyBac Transposases for Genomic Targeting in 

Human Cells 

Jesse B. Owens, Johann Urschitz, Ilko Stoytchev, Stefan Moisyadi. 

570 

Eficacy with Extremely Low Doses of Plasmid DNA 

Using Small Molecule Targeted Delivery of Bilamellar 

Invaginated Vesicles (BIVs) to Melanoma Tumor 

Xenografts 

Nancy S. Templeton, Li Chen, Connor Phalon, Phillip B. Maples, 

Neil Senzer, John Nemunaitis. 

571 

Zinc Finger Nuclease-Based Gene Therapy for Arginase 

I Deiciency as a Model for Curing Single-Enzyme Urea 

Cycle Disorders 

Ragini Bhargava, Hana Park, Sarah Novicoff, Alok V. Joglekar, 

Donald B. Kohn, Gerald S. Lipshutz. 

572 

Poly(2-Dimethylaminoethyl Methacrylate)- 

Functionalized Graphene Oxide Nanolakes for Eficient 

siRNA Delivery 

Jung Eun Lee, Min Sang Lee, Jeong A. Nam, Nak Won Kim, Sung 

Young Park, Ji Hoon Jeong. 

130 




3:45 PM - 5:45 PM 



573 

Eficient Delivery DNA Vaccine for Alzheimer’s Disease 

by Triggered Release of Polyplexes from Microneedles 

Kim Nak Won, Lee Kyuri, Lee Min Sang, Jeong Ji Hoon. 

574 

Improved Lentiviral Formats for Genome Editing Using 

Zinc Finger Nucleases 

Monika Gjoka, Kevin Kang, Yuping Huang, Gregory D. Davis. 

575 

CFTR Reporter Construct for Optimization of Airway 

Epithelium Differentiation 

Myra E. Gordon, Alaleh Esmaeili-Shandiz, Shingo Suzuki, Roy G. 

Sargent, Dieter C. Gruenert. 

576 

Non-Viral Gene Delivery to Oral Squamous Cell 

Carcinoma and Cervical Carcinoma Cells and Suicide 


Nejat Düzgünes, Aruna Singh, Senait Gebremedhin, Krystyna 

Konopka. 

577 

Orphan Product Development for DNA Therapeutics 

Gladice Wallraven, Phillip B. Maples, John Nemunaitis. 



578 

Development of Lentiviral Gene Therapy for Type VII 

Collagen Deicient Epidermolysis Bullosa 

Christos Georgiadis, Ekaterina Semenova, Andy P. South, Adrian 

Thrasher, John A. McGrath, Wei-Li Di, Waseem Qasim. 

579 

Reprogramming of Murine Gallbladder Cells towards 

the Pancreatic b-Cell Fate In Vitro 

Yuhan Wang, Ray D. Hickey, Markus Grompe. 

580 

Fetal Gene Therapy at Early and Late Gestation: 

Analysis of Outcomes and Postnatal Vector Challenge in 

a Macaque Model 

Citra Mattar, Cecilia Rosales, Rowell Fragata, Nuryanti Johana, 

Simon Waddington, Arijit Biswas, Mahesh Choolani, Amit 

Nathwani, Jerry Chan. 

581 

Factors Inluencing Direct Reprogramming of Primary 

Human Extrahepatic Biliary Cells into Pancreatic beta 


Feorillo Galivo, Eric Benedetti, Craig Dorrell, Olga Smirnova, 

Jonathan Schug, Klaus Kaestner, Markus Grompe. 

582 

Mesenchymal Stem Cells Do Not Prevent Alloantibody 

Response Against IDUA Used To Treat MPSI 

Priscila K. Matsumoto, Roberta S. Stilhano, Flavia H. da Silva, 

Vivian Y. Samoto, Giovani B. Perez, Sang W. Han. 

583 

Comparison of Gene Therapy Vectors in a New 

Hypomorphic Model of Propionic Acidemia 

Adam J. Guenzel, Sean E. Hoherr, Matthew Hillestad, Mary Barry, 

Eric Weaver, Jan P. Kraus, Dietrich Matern, Michael A. Barry. 

584 

Transcription Activator-Like Effector Nuclease-Based 

Genome Editing for Mucopolysaccharidosis Type I 

Mark J. Osborn, Colby G. Starker, Anthony P. DeFeo, Daniel F. 

Voytas, Bruce R. Blazar, Jakub Tolar. 

585 

Gene Therapy for Mouse Model of Krabbe Disease 

Hiroshi Kobayashi, Masamichi Ariga, Takahiro Fukuda, Takeo 

Iwamoto, Sayoko Izuka, Yota Shimada, Yoshikatsu Eto, Toya 

Ohashi. 

586 

Choroid Plexus-Directed Viral Gene Therapy as a 

Source of alpha-N-Acetyl-Glucosaminidase Enzyme 

Replacement in Cerebrospinal Fluid for Sanilippo B 

Syndrome 

Patricia I. Dickson, Shih-hsin Kan, Steven Q. Le, Maria Reina 

Haddad, Eun-Young Choi, Anthony Donsante, Stephen G. Kaler. 

587 

Mitigation of DNA Damage Responses and Oxidative 

Stress in Cells with Impaired Telomerase Function 

Larisa Pereboeva, Erik Westin, Mary Gannon, Lawrence Lamb, 

Aloysius Klingelhutz, Frederick Goldman. 

588 Abstract Withdrawn 


589 

CNS Administration of an AAVrh.10 Gene Transfer 

Vector Expressing the Human ARSA cDNA to Nonhuman 

Primates Provides Widespread Activity across the CNS 

Jonathan B. Rosenberg, Dolan Sondhi, David G. Rubin, Sébastien 

Monette, Alvin Chen, Sara A. Cram, Benjamin Van de Graaf, Bishnu 

P. De, Stephen M. Kaminsky, Caroline Sevin, Patrick Aubourg, 

Ronald G. Crystal. 



131




3:45 PM - 5:45 PM 



590 (Moved to Poster Session II after Abstract 391) 

Intracranial AAV Gene Therapy Extends the Life Span 

of GM2 Gangliosidosis Cats >Four-Fold with No Clinical 

Evidence of Vector Toxicity 

Allison M. Bradbury, Victoria J. McCurdy, Aime K. Johnson, Heather 

Gray-Edwards, Brandon L. Brunson, Ashley N. Randle, Nancy R. 

Cox, Miguel Sena-Esteves, Douglas R. Martin. 

591 

Prophylactic and Therapeutic Treatment of Pain 

in a Varicella- Zoster Virus (VZV) Rodent Model of 

Post-Herpetic Neuralgia (PHN) Using an HSV Vector 

Expressing Enkephalin 

William F. Goins, Jean-Marc Guedon, Mingdi Zhang, Joseph C. 

Glorioso, Paul R. Kinchington. 

592 

AAV Gene Therapy Normalizes Disease Phenotype 

and Extends Life Span >Four-Fold in Feline GM1 

Gangliosidosis 

Aime K. Johnson, Victoria J. McCurdy, Allison M. Bradbury, Heather 

Gray-Edwards, Ashley N. Randle, Misako Hwang, Nancy R. Cox, 

Stanley G. Leroy, Miguel Sena-Esteves, Douglas R. Martin. 

593 

MECP2e1 Isoform-Speciic Lentivirus Rescues Rett 

Syndrome Neuronal Soma Size Defects in a Cell 

Autonomous Manner 

Ugljesa Djuric, Aaron Cheung, Alina Piekna, Jason Hendry, Joel 

Ross, Peter Pasceri, Dae-Sung Kim, James Ellis. 

594 

Immune Response Activation and Neurotoxicity after 

Intrathecal Infusion of AAV9 Vector Encoding a Non-Self 

Reporter Protein in the Non-Human Primate 

Lluis Samaranch, Waldy San Sebastian, Adrian P. Kells, John 

Bringas, Phillip Pivirotto, John Forsayeth, Krystof S. Bankiewicz. 

595 

Intracranial Injection of AAVrh8-mbgal in a GM1 

Gangliosidosis Mouse Model Delays Disease Onset and 

Signiicantly Extends Life Span 

Cara M. Weismann, Jennifer Ferreira, Stacy Maitland, Miguel 

Sena-Esteves. 

596 

Enhanced CNS Transduction and Transgene Expression 

by rAAV Capsid Tyrosine Mutants 

Nicholas M. Kanaan, Caryl E. Sortwell, Sara E. Gombash, Nathan 

M. Kuhn, William W. Hauswirth, Sanford E. Boye, Vince A. Chiodo, 

Tessa Grabinski, Fredric P. Manfredsson. 

597 

Peripheral Blood White Cells Present Molecular 

Signatures of Neurodegeneration and Respond to a 

Single Intravenous rAAV9-hNAGLU Gene Delivery in 

MPS IIIB Mice 

Bartholomew J. Naughton, F. Jason Duncan, Tierra Ware, Darren 

Murrey, Aaron Meadows, Douglas M. McCarty, Haiyan Fu. 

598 

The Therapeutic Potential of MicroRNA Regulation in a 

Rat Intracerebral Hemorrhage Model 

Jangsup Moon, Jeong-Min Kim, Jung Ick Byun Byun, Kon Chu, 

Keun-Hwa Jung, Soon-Tae Lee, Jin-Hee Kim, Dong-Kyu Park, So- 

Yoon Kim, Jae-Kyu Roh. 

599 

Adeno-Associated Virus Type 6 Is Retrogradely 

Transported in the Non-Human Primate Brain, 

Implications for Gene Delivery in Huntington’s Disease 

Waldy San Sebastian, Lluis Samaranch, Adrian P. Kells, John 

Bringas, Phillip Pivirotto, John Forsayeth, Krystof S. Bankiewicz. 

600 

Herpes Simplex Virus 2 Gene, ICP10PK, Protects 

Against Neuronal Cell Death Associated with Valproic 

Acid-Induced Autism Spectrum Disorder 

Dominique Bollino, Laure Aurelian. 

601 

Targeted Gene Transfer into the Ependymal Cells 

and Long-Term Enzyme Replacement in the CSF by 

Intraventricular Injection of AAV Vector Serotype 1 

Yoshiyuki Yamazaki, Kohei Hironaka, Yukihiko Hirai, Koichi 

Miyake, Takashi Shimada. 

602 

Neuroprotective Effect of a Cell-Free Extract Derived 

from Human Adipose Stem Cells in Experimental Stroke 

Models 

Jung Ick Byun, Daejong Jeon, Kon Chu, Soon-Tae Lee, Keun-Hwa 

Jung, Jae-Jun Ban, Hye-Jin Yoon, Seungmoon Jung, Hyunwoo Yang, 

Byung Sun Kim, Ji Ye Choi, Jeong-Min Kim, Chong-Hyun Won, 

Manho Kim, Sang Kun Lee, Jae-Kyu Roh. 

603 

Artiicial MicroRNAs Against Spliced Variants of 

the Gene C9ORF72, the Major Cause for Familial 

Amyotrophic Lateral Sclerosis 

Gabriela Toro, Florie Borel, Peter Sapp, Lina Song, Robert Brown, 

Christian Mueller. 

604 

Reduction of Hyperammonemia In Vitro and In Vivo by 

a Baculovirus-Delivered Glutamine Synthetase as a New 

Approach for the Treatment of Hepatic Encephalopathy 

Miguel A. Torres-Vega, Lizbeth M. Vázquez-Carcaño, Roxana 

Y. Vargas-Jerónimo, Ana G. Montiel-Martínez, Rosa M. Muñoz- 

Fuentes, Misael Uribe, Absalom Zamorano-Carrillo, Ana R. Pastor- 

Flores, Laura A. Palomares. 

132 




3:45 PM - 5:45 PM 



605 

MTMR2 Ameliorates the Phenotype of Myotubular 

Myopathy in Mice 

Nathalie Danièle, Thibaud Jamet, Christelle Moal, Romain Joubert, 

Nadia Messaddeq, Michael W. Lawlor, Alban Vignaud, Anna 

Buj-Bello. 

606 

Restricting MTM1 Transgene Expression to Skeletal 

Muscle in AAV-Mediated Gene Therapy for Myotubular 

Myopathy 

Romain Joubert, Christelle Moal, Alban Vignaud, Samia Martin, 

Isabelle Richard, Philippe Moullier, Alan H. Beggs, Martin K. 

Childers, Anna Buj-Bello. 

607 

Long Term Intra-Articular Expression of PRG4 Prevents 

Post-Traumatic Osteoarthritis 

Zhechao Ruan, Vincenzo Cerullo, Kilian Guse, Ayelet Erez, Brian 

Dawson, Frank Gannon, Brendan Lee. 

608 

Immune Tolerance Induction in Canine X-Linked 

Muscular Dystrophy with Trans-Placental rAAV9- 

Microdystrophin Transduction 

Hiromi Hayashita-Kinoh, Hironori Okada, Yuko Nitahara-Kasahara, 

Tomoko Chiyo, Naoko Yugeta, Takashi Okada, Shin’ichi Takeda. 

609 

Low Dystrophin Levels Increase Survival and Improve 

Muscle Pathology and Function in Dystrophin/Utrophin 

Double Knockout Mice 

Maaike van Putten, Margriet Hulsker, Courtney Young, Peter A. ‘t 

Hoen, Gert-Jan B. van Ommen, Annemieke M. Aartsma-Rus. 

610 

Limb Girdle Muscular Dystrophy 2I: Generation of a 

New Mouse Model and Test of Therapeutic Approaches 

by rAAV2/9 Gene Transfer 

Evelyne Gicquel, Karine Charton, Nathalie Daniele, Isabelle 

Richard. 

611 

Hepatocyte Growth Factor Contributes to the Improved 

Pathology of mdx:p65+/- Mice 

Jonathan D. Proto, Ying Tang, Aiping Lu, Minakshi Poddar, Sarah 

Beckman, Kayla Imbrogno, Timothy Hannigan, Wendy Mars, Bing 

Wang, Johnny Huard. 

612 

Urine Biomarkers for Duchenne Muscular Dystrophy 

and Its Animal Models 

Jérémy Rouillon, Thibaut Léger, Jean-Michel Camadro, Tomas Voit, 

Fedor Svinartchouk. 

613 

Systemic Gene Transfer in Neonatal Dogs Using 

Tyrosine Mutant AAV-1,6 and9 

Chady H. Hakim, Yongping Yue, Jin-Hong Shin, Regina R. Williams, 

Keqing Zhang, Arun Srivastava, Bruce F. Smith, Dongsheng Duan. 

614 

Effect of Angiogenesis on the Regenerative Capacity of 

ACL-Derived CD34+ Cells in ACL Reconstruction 

Koji Takayama, Tang Ying, Wang Bing, Freddie Fu, Johnny Huard. 

615 

The Phenotype of Dysferlin-Deicient Mice Is Not 

Rescued by AAV-Mediated Transfer of Anoctamin 5 

Karine Charton, Laurence Suel-Petat, François Monjaret, Nathalie 

Bourg-Alibert, Carinne Roudaut, Evelyne Gicquel, Bjarne Udd, 

Isabelle Richard. 

616 

Transfection of Murine Dermis Via Topical DNA 

Plasmid Delivery 

Chunqing Dou, Guy P. Marti, Lixin Liu, Frank Lay, Donald J. Rees, 

Aaron T. Tabor, Junkai Du, Sayed Mohammd Hosseini, Amir Mehdi 

Ansari, Ali Karim Ahmed, James A. Williams, John W. Harmon. 

617 

Clinical Trial Readiness for Gene Replacement to the 

Diaphragm: Translational Respiratory Endpoints in 

Ventilator-Dependent Neuromuscular Diseases 

Barbara K. Smith, Melissa A. Goddard, Martin K. Childers, Barry J. 

Byrne. 

618 

Adjunctive b2-Agonist Therapy Enhances Traficking of 

Acid a-Glucosidase to Lysosomes in the Skeletal Muscle 

of Mice with Pompe Disease 

Dwight D. Koeberl, Songtao Li, Benjamin Farah, Andrew Bird, Paul 

M. Yen, Sarah P. Young. 

619 

Comparative In Vitro Potency of a Novel Disc-Derived 

Cell Population (Discospheres) and Mesenchymal Stem 

Cells for the Treatment of Degenerative Disc Disease 

Lara I. Silverman, Galina Dulatova, Terry Tandeski, Kevin T. Foley. 

620 

Functional Analysis of GNE Expression Plasmids in 

Immortalized Human Myoblasts 

Yadira Valles-Ayoub, Rosangela Carbajo, Lucia Sandoval, Jorge 

Garcia-Figueroa, Sarah Stein, Zeshan Khokher, Daniel No, Babak 

Darvish, Jacques P. Tremblay, Daniel Darvish. 

Cancer Immunotherapy III 

621 

Tethered IL-15 Mutein on CD19-Speciic T Cells 

Sustains Persistence When Tumor Antigen Is Low and 

Can Treat Minimal Residual Disease 

Lenka V. Hurton, Kirsten Switzer, Harjeet Singh, Marie A. Forget, 

Tiejuan Mi, Simon Olivares, Richard E. Champlin, Dean A. Lee, 

Laurence J. N. Cooper. 



133




3:45 PM - 5:45 PM 


Cancer Immunotherapy III 

622 

Rapid Assessment of T-Cell Function in an Animal-Free 

Model – The Artiicial-Mouse Model 

Usanarat Anurathapan, Pradip Bajgain, Norihiro Watanabe, 

Malcolm K. Brenner, Cliona M. Rooney, John R. Wilson, Juan F. Vera. 

623 

Distinct Signaling from Chimeric Antigen (CAR) and T 

Cell Receptors in T Cells 

Omkar U. Kawalekar, Avery D. Posey Jr, Sonia Guedan, Jihyun Lee, 

Matthew J. Frigault, Maria C. Basil, John Scholler, Yangbing Zhao, 

Carl H. June. 

624 

Long Term F/U – Phase I Study of the “Triad” 

Autologous (FANG) Vaccine, Incorporating 

Bifunctional shRNAfurin and GMCSF Transgene 

Expression, in Advanced Cancer Patients 

John Nemunaitis, Neil Senzer, Minal Barve, Cynthia Bedell, Joseph 

Kuhn, Peter Beitsch, Robert Steckler, Jeffrey Lamont, Robert 

Hebeler, Mitchell J. Magee, Walton Taylor, Brian M. Gogel, Maurizio 

Ghisoli, F. Charles Brunicardi, Beena O. Pappen, Padmasini Kumar, 

Phillip B. Maples. 

625 

Phase I Study Results of pbi-shRNA STMN1 Nanoplex 

Via Intratumoral Injection in Advanced Cancer 

John Nemunaitis, F. Charles Brunicardi, Donald D. Rao, Nancy S. 

Templeton, Neil Senzer, Zhaohui Wang, Minal Barve, Padmasini 

Kumar, Gladice Wallraven, Christopher M. Jay, Joseph Kuhn, Peter 

Beitsch, Phillip B. Maples. 

626 

T-Cell Immunotherapy for Multiple Myeloma 

Sonny O. Ang, Sourindra N. Maiti, Ippei Sakamaki, Hong Qin, Larry 

W. Kwak, Richard E. Champlin, Laurence J. N. Cooper. 

627 

Gene Transfer of Chondroitin Sulfate-Coated 

Granulocyte Macrophage-Colony-Stimulating Factor in 

Mouse Ovarian Tumor Model 

Katsuyuki Hamada, Kazuko Takagi, Chieko Yoshihara, Tomoko Ito, 

Yoshiyuki Koyama, Hiroshi Ito, Akihiro Nawa. 

628 

EphrinA1-EphA2 Interaction-Mediated Apoptosis and 

Flt3L-Induced Immunotherapy in Inhibiting Tumor 

Progression in an Immunocompetent Mouse Model of 

Breast Cancer 

Sai vikram V. vemula, Manish Tandon, Suresh K. Mittal, Anurag 

Sharma, Dinesh Bangari. 

629 

Optimization of Chimeric Antigen Receptors Design: 

Novel Retroviral Vectors Expressing CAR with 

Intracellular Signaling Domain of Glucocorticoid- 

Induced TNF-Receptor 

Yasunori Amaishi, Sachiko Okamoto, Yoshikazu Kurosawa, Hiroshi 

Shiku, Junichi Mineno, Kazuto Takesako. 

630 

Combined p19Arf and Interferon-beta Gene Therapy: 

Evidence of Immune Response in Murine Models of 

Melanoma and Lung Carcinoma 

Bryan E. Strauss, Ruan Felipe V. Medrano, Aline H. Ribeiro, Joao 

Paulo P. Catani, Christian A. Merkel. 

631 

Histidine-Rich Glycoprotein (HRG): A Novel Gene- 

Therapy Effector for the Treatment of Cancer 

Francis P. Roche, K. Elisabet Ohlin, Magnus Essand, Lena 

Claesson-Welsh. 

632 

Effect of Interferon-Omega Gene Transfer on Feline 

Mammary Carcinoma and Melanoma Cells 

Marcela S. Villaverde, Alexandra M. Targovnik, Daniel R. Bafico, 

Maria V. Miranda, Gerardo C. Glikin, Liliana M. E. Finocchiaro. 

633 

HLA Typing Identity Test for Patients and Their FANG 

Autologous Cancer Vaccines – Update 

Yang Yu, Padmasini Kumar, Fabienne Norvell, Connor Phalon, 

Nicolas Taquet, Phillip B. Maples. 


634 

Therapeutic Targeting of Chitosan-PEG-Folate- 

Complexed Oncolytic Adenovirus for Active and 

Systemic Cancer Gene Therapy 

Oh-Joon Kwon, Eunah Kang, Sung Wan Kim, Chae-Ok Yun. 

635 

Human Ovarian Cancer-Derived Ascitic Fluid Has Mixed 

Effects on CRAds Eficacy 

Veronica M. Lopez, Nicasio Cuneo, Mariela A. Gangemi, Leonardo 

Sganga, Marina Demonte, Alejandro Soderini, Osvaldo L. 

Podhajcer. 

636 

The Oncolytic Virus DPK Lyses Cells with Cancer 

Stem Cell (CSC) Properties through Calpain-Mediated 

Clearance of the Selective Autophagy Protein p62/ 

SQSTM1 

Aric Colunga, Dominique Bollino, Amanda Schech, Laure Aurelian. 

637 

Demonstration of Anti-Metastasis Activity of Oncolytic 

Measles Virus Retarging Urokinase Receptor in 

Experimental Breast Cancer Metastasis Model 

Yuqi Jing, Krisztina Kovacs, Jaime Merchan. 

134 




3:45 PM - 5:45 PM 



638 

Development of Dual Targeted Oncolytic Adenovirus 

for Human Malignant Mesothelioma 

Shuji Kubo, Misato Takagi-Kimura, Atsuko Tamamoto, Tomoko 

Hashimoto-Tamaoki, Noriyuki Kasahara, Masatoshi Tagawa. 

639 

MicroRNA-Mediated Multi-Tissue-Detargeting of 

Oncolytic Measles Virus 

Marc-Andrea Bärtsch, Mathias F. Leber, Sascha Bossow, 

Martin Singh, Jessica Albert, Dirk Jäger, Christof von Kalle, Guy 

Ungerechts. 


647 

Iraqi Newcastle Disease Virus Virulent Strain as Cancer 

Antiangiogenic Agent 

Ahmed M. Al-Shammari, Zaid A. Al-Hili, Nahi Y. Yaseen. 

Cancer-Targeted Gene & Cell Therapy: Cell 

Therapy / Immunotherapy / Cellular Targets 

648 

Neural Stem Cells Expressing a Modiied Human 

Carboxylesterase Potentiate Anti-Cancer Activity of 

Irinotecan in Murine Glioma Model 

Karen S. Aboody, Lucy Y. Ghoda, Margarita Gutova, Megan 

Gilchrist, Christine Brown, Vazgen Kankaldyyan, Elizabth Garcia, 

Lauren Hoffman, Rex A. Moats, Phil Potter, Timothy W. Synold, 

Michael Barish. 

640 

In Vivo Therapeutic Eficacy of Oncolytic Adenovirus 

Delivered by New Biodegradable Polymers through 

Systemic Injection 

Jaesung Kim, Yi Li, Sung Wan Kim, Doo Sung Lee, Chae-Ok Yun. 

641 

Eficacy of Measles Virotherapy in Osteosarcoma 

Evidio Domingo Musibay, Cory Allen, Ianko D. Iankov, Jayson J. 

Hardcastle, Evanthia Galanis. 

642 

A Cancer-Targeting Ligand Strongly Enhances Oncolytic 

Activity of a Conditionally Replicative Adenovirus 

Yuki Yamamoto, Naoko Goto, Shumpei Ohnami, Masatoshi Tagawa, 

Kazunori Aoki. 

643 

Cyclophosphamide Enhances Antitumor 

Eficacy of Oncolytic Adenovirus Expressing 

Uracil Phosphoribosyltransferase (UPRT) in 

Immunocompetent Syrian Hamsters 

Naoyuki Hasegawa, Masato Abei, Kazunari K. Yokoyama, Kuniaki 

Fukuda, Emiko Seo, Rei Kawashima, Yuri Nakano, Takeshi Yamada, 

Koji Nakade, Hirofumi Hamada, Yuichi Obata, Ichinosuke Hyodo. 

644 

Therapeutic Evaluation of Prostate Cancer Speciic 

Oncolytic Adenovirus Armed with Fusion Protein Gene 

PSA-IZ-CD40L 

Yue-feng Yang, Shu-Ya Xue, Hua Wang, Feng-jun Xiao, Yue Yin, 

Zhuo-zhuang Lu, Qun-wei Zhang, Chu-tse Wu, Li-sheng Wang. 

645 

Bioreducible Polymer-Conjugated Oncolytic 

Adenovirus for Hepatoma-Speciic Therapy Via 

Systemic Administration 

Pyung-Hwan Kim, Jaesung Kim, Tae-il Kim, Hye Yeong Nam, James 

W. Yockman, Minjung Kim, Sung Wan Kim, Chae-Ok Yun. 

649 

Re-Directing T Cells with Chimeric Antigen Receptors 

To Target CD123+ Leukemia 

Radhika Thokala, Kirsten Switzer, Mi Ti juan, Simon Olivares, 

Harjeet Singh, Helen Huls, Richard E. Champlin, Laurence J. N. 

Cooper. 

650 

The Speciicity of Rhesus T Cells Can Be Redirected 

to CD20 Using the Sleeping Beauty Transposon/ 

Transposase System To Express a Chimeric Antigen 

Receptor 

Simon Olivares, Colleen O’Connor, Sonny Ang, Leslie Kean, Perry 

Hackett, Amer Najjar, Harjeet Singh, Helen Huls, Elizabeth Shpall, 

Laurence Cooper. 

651 

Tumor Targeting Using CD19-Speciic CAR (Chimeric 

Antigen Receptor) for B-Cell Lymphoma 

Tomonori Tsukahara, Ken Ohmine, Ryosuke Uchibori, Hiroyuki 

Ido, Masashi Urabe, Hiroaki Mizukami, Akihiro Kume, Masataka 

Nakamura, Junichi Mineno, Kazutoh Takesako, Isabelle Riviere, 

Michel Sadelain, Renier Brentjens, Keiya Ozawa. 

652 

Tumor Relapse Is Associated with an Innate Immune 

Resistant Phenotype across Tumor and Treatment 

Types Which Can Be Targeted with Rational Second 

Line Therapies 

Timothy Kottke, Oliver Donnelly, Nicolas Boisgerault, Rosa 

Diaz, Diana Rommelfanger-Konkol, Jose Pulido, Jill Thompson, 

Debabrata Mukhopadhyay, Keith Knutson, Marshall Behrens, 

Roger Kaspar, Matt Coffey, Peter Selby, Kevin Harrington, Alan 

Melcher, Hardev Pandha, Richard Vile. 

653 

Chimeric Antigen Receptor T-Cell Therapy for 

Companion Canines with Spontaneous B-Cell Non- 

Hodgkin Lymphoma 

Colleen M. O’Connor, Simon Olivares, Sonny Ang, Richard E. 

Champlin, Heather M. Wilson-Robles, Laurence J. N. Cooper. 



135




3:45 PM - 5:45 PM 


Cancer-Targeted Gene & Cell Therapy: Cell 

Therapy / Immunotherapy / Cellular Targets 

654 

Combination of CD3+CD56+ (NKT) Cells 

Immunotherapy and Adenovirus-p53 Gene Therapy for 

Head and Neck Squamous Cell Carcinoma 

Hiroki Saito, Naoya Morishita, Kyung-Mi Lee, Dante Dator, Masato 

Kawabata, Masato Fujisawa, Toshiro Shirakawa. 

655 

Interfering Peptides Targeting Transcription Factor 

AP1 for Pancreatic Cancer Gene Therapy 

Soukaina Rejiba, Megan Frelan, Alex Hermann, Katja Arndt, Amor 

Hajri. 

656 

JMJD3 Regulates Senescence-Associated Secretory 

Phenotype Factors Responsible for Robust Neural Stem 

Cell Tropism toward Glioma 

Michael E. Silva, Patrick M. Perrigue, Daniel Mota, Lauren Joseph, 

Nathan Feng, Michael Reid, Joseph Najbauer, Karen S. Aboody. 

657 

Bioengineering ACE Synthetic Chromosomes for Anti- 

Cancer Gene Therapy 

Edward Perkins, Jon Holy, Teresa Rose-Hellkant, Amy Greene. 

658 

DNA Vaccine with scFv6.C4 Prevent the Growth of 

Tumor Expressing CEA 

Priscila M. A. Denapoli, Roberta S. Stilhano, Priscila K. Matsumoto, 

Carolina B. Parise, Jane Z. de Moraes, Wolfgang Zimmermann, Sang 

W. Han. 

659 

Presumptive Oncogenic Transcriptional Factor PDX1 

Regulates Pancreatic Cancer Via an Ampliication Loop 

Involving the RAS, PI3K and p53 Signaling Pathways 

Juehua Yu, Guisheng Zhou, Shi-he Liu, James Wu, John Nemunaitis, 

Neil Senzer, Donald Rao, Enrique Rosengurt, F. C. Brunicardi. 

660 

Targeting Lysosome Integrity as a Method To Restore 

Sensitivity to Ceramide-Induced Cell Death 

Lorianne S. Turner, Krissy Smith, Lenton Holley, Christopher 

Johnson, Timothy Prince, Heather Yancey. 


Therapy II 

661 

Lentiviral Gene Therapy of RAG Severe Combined 


Niek P. van Til, Guus van der Velden, Helen de Boer, Pietro L. 

Poliani, Michael N. Antoniou, Anna Villa, Chantal Lagresle-Peyrou, 

Marina Cavazzana-Calvo, Fang Zhang, Adrian J. Thrasher, Gerard 

Wagemaker. 

662 

Stem Cell and T-Cell Gene Therapy Using SIN- 

Lentiviral Vector in Type 3 Familial Hematophagocytic 

Lymphohistocytosis: A Comparative Approach 

Julie Rivière, Tayebeh Soheili, Els Verhoeyen, Anne Galy, Geneviève 

De Saint Basile, Marina Cavazzana-Calvo. 

663 

High Expression Bioengineered Factor VIII Enables 

AAV-Based Gene Therapy of Hemophilia A 

H. Trent Spencer, Harrison C. Brown, Jordan E. Shields, J. Fraser 

Wright, Christopher B. Doering. 

664 

Identiication of Novel Combination of Elements in 

cHS4 with Optimal Insulator Activity and Improved 

Titers in g-Globin Carrying SIN Lentiviral Vectors 

Paritha Arumugam, Michael Wourms, Joseph Starnes, Catherine 

Fox, Xiaoming Li, Punam Malik. 

665 

Intraosseous Delivery of Lentiviral Vectors Expressing 

Factor VIII under the Control of the Platelet-Speciic 

Glycoprotein 1ba Promoter Leads to Long-Term 

Correction of Bleeding in Hemophilia A Mice 

Xuefeng Wang, Andy Chiang, Simon C. Shin, Liping Chen, Dao Pan, 

David J. Rawlings, Carol H. Miao. 

666 

Lentiviral Vectors Tailored for Liver-Directed Gene 

Therapy Do Not Show Any Detectable Genotoxicity in 

Sensitive In Vivo Biosafety Assays 

Marco Ranzani, Alessio Cantore, Monica Volpin, Pierangela Gallina, 

Stefano Annuziato, Francesca Sanvito, Fabrizio Benedicenti, 

Cynthia Bartholomae, Lucia Sergi Sergi, Christof von Kalle, 

Manfred Schmidt, Claudio Doglioni, Luigi Naldini, Eugenio 

Montini. 

667 

Successful Liver Gene Therapy of Hemophilia B 

Dogs Using Lentiviral Vectors and Hyperfunctional 

Transgene 

Alessio Cantore, Patrizia Della Valle, Sara Bartolaccini, Lucia Sergi 

Sergi, Thierry VandenDriessche, Marinee Chuah, Dwight Bellinger, 

Armando D’Angelo, Timothy Nichols, Luigi Naldini. 

136 




3:45 PM - 5:45 PM 



Therapy II 


733 

Correction of canine Factor VII (FVII) deiciency by 

AAV-mediated expression of canine FVII zymogen 

Paris Margaritis, Dwight A. Bellinger, Shangzhen Zhou, Armida 

Faella, Timothy C. Nichols, Katherine A. High 

668 

Engineering of a HIV-Resistant Immune System with 

Hematopoietic Stem Cells – A Polycistronic Approach 

Janet Chung, Chy-Anh Tran, Angel Gu, Lisa Scherer, Haitang Li, 

David L. DiGiusto, John J. Rossi. 

669 

Direct and Indirect Immune Suppressive Effects of High 

Hepatic Expression Direct Effective Reversal of Factor 

IX Inhibitors by AAV Gene Transfer 

David Markusic, Roland Herzog. 

670 

Combination Therapy of Anti-CD20, AMD3100, G-CSF 

and Factor VIII Plasmid-Mediated Gene Therapy 

Can Successfully Promote Tolerance to Factor VIII in 

Hemophilia A Mice with Pre-Existing Inhibitors 

Chao Lien Liu, Meghan Lyle, Simon C. Shin, Carol H. Miao. 

671 

Identiication of Novel Chromatin Insulator 

Elements for Use in Lentiviral Gene Therapy of T Cell 

Immunodeiciencies 

Sheng Zhou, Taihe Lu, Zhijun Ma, John T. Gray, Brian P. Sorrentino. 

672 

Transduction of Human CD34+ Cells in the NOD SCIDg 

(NSG) Mouse Model Using Lenti-hWASP-Vectors Derived 

from Stable Producer Clones 

Matthew M. Wielgosz, Yoon S. Kim, Robert E. Throm, Gael G. 

Carney, John T. Gray, Arthur W. Nienhuis. 

673 

Pre-Clinical Modeling of Foamy Virus Vector Based 

Gene Therapy for SCID-X1 

Gabrielle M. Curinga, Iram Khan, Swati Singh, Joey Pangallo, Brian 

Beard, Troy Torgerson, Andrew Scharenberg, Grant Trobridge, 

Hans-Peter Kiem, David Rawlings. 

674 

Long Term Safety of Clinical Grade LentiGlobin Vectors 

in a b-Thalassemic and Normal Mice 

Olivier Negre, Cynthia Bartholoma, Robert Kutner, Beatrix Gillet- 

Legrand, Celine Courne, Anais Paulard, Byoung Ryu, Maria Denaro, 

Christof von Kalle, Emmanual Payan, Michell Finer, Gabor Veres. 



735 

Optimization of dual-function vectors for Alpha-1 

Antitrypsin Deiciency 

Qiushi Tang, Christian Mueller, Lina Song, Terence Flotte 

736 

Targeting dendritic cells with lentiviral vectors induces 

tolerance of effector T cells and protects mice from 

experimental autoimmune encephalomyelitis 

Bruna de Andrade Pereira, Mathias Ackermann, Christiane Dresch, 

Cornel Fraefel 

737 

Novel adenoviral vectors induce robust T cell responses 

to HSV2 and signiicantly boost responses after repeat 

homologous administration 

Christopher A. Lazarski, Douglas E. Brough, Lisa L. Wei 

738 

TALEN-based Individualized Gene Medicine for 


Mark J. Osborn, Colby G. Starker, Amber N. McElroy, Beau R. 

Webber, Anthony P. DeFeo, Richard Gabriel, Manfred Schmidt, 

Christof von Kalle, Daniel F. Carlson, Morgan L. Maeder, J. Keith 

Joung, John E. Wagner, Daniel F. Voytas, Bruce R. Blazar, Jakub 

Tolar 

739 

Prevention of Anti-Capsid Immune Response Following 

Systemic AAV9 Dosing in Infant Rhesus Monkeys 

Darin Falk 

740 

Inhibition of ocular neovascularization by 

subconjunctival gene delivery of calreticulin-derived 

peptide, vasostatin 112 

Guei-Sheung Liu, Youn-Shen Bee, Gregory J. Dusting, Ming-Hong 

Tai 

741 

Transient Epigenetic Gene Therapy as a New 

Therapeutic/Prophylactic Avenue for Aging/ 

Degenerative Diseases and Neurodevelopmental 

Disorders 

Roger Bertolotti 

742 

Successful Systemic Cancer Targeting by Adenovirus 

Vector with AB-loop Redesigned Fiber via Reducing 

Liver Sequestration 

Yoshiaki Miura, Joohee Han, Julia Davydova, Masato Yamamoto 



137



3:45 PM - 5:45 PM 



743 

Prevention of radiation-induced salivary hypofunction 

by delivering hSFRP2 into submandibular glands 

Changyu Zheng, Ana P. Cotrim, Corinne Goldsmith, Stefanie 

Aquilina, Anastasia Sowers, Indu S. Ambudkar, James B. Mitchell, 

Bruce J. Baum 

744 

The non-signaling extracellular spacer domain of 

CD19-speciic chimeric antigen receptors is decisive for 

in vivo antitumor activity 

Michael Hudecek, Lingfeng Liu, Anne Silva, Paula Kosasih, Yvonne 

Y. Chen, Cameron J. Turtle, Michael C. Jensen, Stanley R. Riddell 


746 

Determining the physiologic effect of B-Type 

Natriuretic Peptide on cardio-renal homeostasis 

Sara J Holditch, Alessandro Cataliotti, Aron Geurts, Yasuhiro Ikeda 

747 

Bortezomib induced unfolded protein response 

synergizes with oHSV to induce necroptic cell death in 

solid tumors 

Ji Young Yoo, Brian Hurwitz, Chelsea Bolyard, Jun-Ge Yu, Jeffrey 

Wojton, Jianying Zhang, Matthew Old, Balveen Kaur 

753 

Non-myeloablative preconditioning with Ack-2 for cell 

therapy for Hunter disease 

Takayuki Yokoi, Kentaro Yokoi, Kazumasa Akiyama, Takashi 

Higuchi, Yohta Shimada, Hiroshi Kobayashi, Yoshikatsu Eto, Taku 

Sato, Makoto Otsu, Hiromitsu Nakauchi, Shinichi Nishikawa, 

Hiroyuki Ida, Toya Ohashi 

754 

Human placenta-derived mesenchymal stem cells 

augments spinal cord functional recovery in scid mice 

model 

Vikram Sabapathy, Dorai Murugan, George Tharion, Sanjay Kumar 

755 

Correlation of molecular parameters with lipoplex 

structure and transfection eficacy in pyridinium-based 

cationic lipids 

Paria Parvizi, Emile Jubeli, Liji Raju, Nada Abdul Khalique, Ahmed 

Almeer, Hebatalla Allam, Maryem Al Manaa, Helge Larsen, David 

Nicholson, Michael D. Pungente, Thomas M. Fyles 

756 

Transplantation of human adipose tissue-derived stem 

cells provides behavioral improvement, clinical onset 

delay and life extension in mouse 

Hong J. Lee,Jin An, Kwang S. Kim, In J. Lim, Seung U. Kim 

748 

Assessing Small Vector Pharmacology Using the Cre- 

LoxP System in Mice 

Matthew L. Hillestad, Adam Guenzel, Michael A. Barry 


750 

Hydrodynamic Delivery of mIL10 Gene Protects Mice 

from High-Fat Diet-induced Obesity and Glucose 

Intolerance 

Mingming Gao, Chunbo Zhang, Yongjie Ma, Le Bu, Linna Yan, Dexi 

Liu 


751 

Heparin afinity puriication of extracellular vesicles 

Leonora Balaj, Nadia A. Atai, Weilin Chen, Dakai Mu, Bakhos A. 

Tannous, Johan Skog, Xandra O. Breakeield, Casey A. Maguire 

752 

Preclinical study of AAV-sTRAIL: pharmaceutical 

eficacy, tissue distribution, and animal safety 

Dexian Zheng, Xin Wang, Wei Li, Yanxin Liu 

138 


Abstract Author Index 

Aartsma-Rus, Annemieke M. ........................................................609 

Abate-Daga, Daniel ...........................................................................716 

Abdalla, Renne J. ................................................................................265 

Abdul-Razak, Hayder H ..................................................................678 

Abei, Masato ........................................................................................643 

Abels, Erik R. .......................................................................................304 

Abernathy, Corinne .................................................................. 55, 721 

Abhyankar, Harshal ..........................................................................305 

Aboody, Karen S. ......................................................................648, 656 

Abramihina, Tatiana .........................................................................465 

Abuin, Allison M....................................................................................31 

Acchione, Mauro ................................................................................477 

Ackermann, Mania ...............................................................................84 

Ackermann, Mathias ........................................................................736 

Adachi, Kei ........................................................72, 78, 119, 124, 259 

Adair, Jennifer E. ........................................................................ 25, 421 

Adamo, Richard F. ...................................................................366, 372 

Adams, Katherine ..............................................................................508 

Aderca, Ileana ........................................................................................17 

Adjali, Oumeya ...................................................................................111 

Agapova, Larisa ........................................................................137, 470 

Agarwal, Prachi ..................................................................................537 

Agbandje-McKenna, Mavis ................. 4, 71, 333, 334, 335, 541 

Aghi, Manish K. ...................................................................................676 

Aguiar, Rodrigo ..................................................................................519 

Aguilar, Tania ......................................................................................476 

Ahmed, Ali Karim ..............................................................................616 

Ahmed, Seemin S. .................................................................................35 

Ahonen, Marko ...................................................................................501 

Aiuti, Alessandro ......................................................28, 63, 309, 517 

Ai-Young, Lee .......................................................................................699 

Akbarpour, Mahzad ................................................................323, 441 

Akbuga, Jülide .....................................................................................452 

Akinfenwa, Patricia Y. ......................................................................131 

Akiyama, Kazumasa ...............................................................365, 753 

Akpek, Gorgon ....................................................................................298 

Al Manaa, Maryem ............................................................................755 

Alan, Saadet .........................................................................................452 

Albert, Jessica .....................................................................................639 

Albert, Lee ............................................................................................471 

Alberti, Michael O. .............................................................................438 

Alexander, Ian E. ................................................................................356 

Alexandrov, Krill ................................................................................224 

Alferiev, Ivan........................................................................................372 

Al-Hili, Zaid A. .....................................................................................647 

Allam, Hebatalla.................................................................................755 

Allen, Cory .......................................................................... 23, 417, 641 

Allen, James M. ...................................................................................543 

Allen, Margaret D. ................................................................................96 

Almeer, Ahmed ...................................................................................755 

Almeida-Porada, Graca ...................................................................253 

Alonso-Ferrero, María Eugenia ...................................................678 

Al-Shammari, Ahmed M. ................................................................647 

Altaleb, Naderah ................................................................................379 

Alusi, Ghassan .....................................................................................561 

Amaishi, Yasunori ..................................................................... 16, 629 

Amano, Shinya U. ..................................................................................96 

Amante, Dinah ....................................................................................153 

Amante, Romain ................................................................................458 

Amarante, Trish .................................................................................339 

Ambudkar, Indu S. .............................................................................743 

Ammayappan, Arun .........................................................................413 

An, Dong Sung ....................................................................................530 

An, Jin .....................................................................................................756 

An, Songtao ..........................................................................................369 

Anagnostopoulos, Achilles .................................................... 27, 427 

Andino, Lourdes M. ..........................................................................542 

Ando, Dale ...............................................................................................58 

Ando, Miki ............................................................................................684 

Andre, Franck M. ...............................................................................148 

Ang, Sonny ............................................................ 198, 626, 650, 653 

Angelino, Mark D. ....................................................................260, 262 

Anguela, Xavier M. ..................................................................110, 488 

Ankoudinova, Irina ...........................................................................184 

Anna, Stornaiuolo .............................................................................520 

Annan, Anand C..................................................................................438 

Annoni, Andrea ........................................................................323, 441 

Annunziata, Patrizia .........................................................................362 

Annunziato, Stefano ...............................................................233, 666 

Ansari, Amir Mehdi ..........................................................................616 

Anstaett, Olivia ...................................................................................185 

Antico, Christopher J........................................................................324 


139


Antignac, Corinne..............................................................................546 

Antoniou, Michael N. ..............................................................430, 661 

Anurathapan, Usanarat ........................................................398, 622 

Anwer, Khursheed ............................................................................461 

Aoki, Kazunori ..........................................................................414, 642 

Aouadi, Myriam.....................................................................................96 

Aprile, Annamaria .............................................................................420 

Aquilina, Stefanie ..............................................................................743 

Aramaki, Yukihiko .............................................................................142 

Arambula, Claudia ............................................................................394 

Aras, Rahul ...........................................................................................500 

Arbeit, Jeffrey M. ................................................................................560 

Arbel-Ornath, Michal .......................................................................283 

Arber, Caroline ...................................................................................305 

Arden, Erik ...........................................................................................538 

Ardinger, Jeffery .................................................................................338 

Arens, Anne .........................................................................................204 

Ariga, Masamichi ...............................................................................585 

Armentano, Donna ...........................................................................338 

Arndt, Katja .........................................................................................655 

Arndt, Michaela A. E. ...........................................................................21 

Arnold, Eric ..........................................................................................454 

Arnould, Sylvain .................................................................................401 

Aronovich, Elena L. .................................................................102, 447 

Aronowitz, Eric .....................................................................................11 

Arpke, Robert W.................................................................................168 

Arruda, Valder R. ...............................................................................498 

Artus, Alexandre ...................................................................................64 

Arumugam, Paritha ..........................................................................664 

Arumugam, Ramamani ...................................................................276 

Arya, Suresh ..............................................................................531, 696 

Asaad, Wael..........................................................................................387 

Aschbacher, Kirstin A. .....................................................................369 

Ashley, Scott ........................................................................................108 

Ashtari, M. ...............................................................................................53 

Aslanidi, George ............................................................ 333, 334, 541 

Aslanidi, George V. ......................................75, 118, 314, 328, 542 

Asokan, Aravind ....................................................................... 2, 73, 77 

Aswani, Vijay H. .................................................................................280 

Atai, Nadia A. .......................................................................................751 

Atala, Anthony ....................................................................................253 

Athanasopoulos, Takis ....................................................................734 

Atherton, Pamela ..................................................................................17 

Aubourg, Patrick ....................................................62, 278, 279, 589 

Aucher, Anne ..........................................................................................24 

Aurelian, Laure .........................................................................600, 636 

Auricchio, Alberto .............................................. 340, 359, 360, 499 

Badr, Christian E. ...............................................................................458 

Badros, Ashraf Z. ...............................................................................298 

Bae, You Han ........................................................................................556 

Baek, Sun-Young ................................................................................375 

Baert, Miranda R. M. ........................................................................313 

Bafico, Daniel R. ................................................................................632 

Bagel, Jessica .......................................................................................289 

Baird, Michelle....................................................................................255 

Bajgain, Pradip ...................................................................................622 

Bake, Erin .............................................................................................712 

Baker, David ...............................................................................188, 318 

Balaj, Leonora .....................................................................................751 

Balakrishnan, Amritha ............................................................ 45, 397 

Balakrishnan, Balaji .........................................................................337 

Baliakas, Panayotis ...........................................................................427 

Ballon, Douglas J. ..................................................................................11 

Balzer, Frederick ...................................................................................92 

Ban, Jae-Jun ..........................................................................................602 

Bangari, Dinesh ..................................................................................628 

Bankiewicz, Krystof S. ...................................... 288, 594, 599, 703 

Bao, Gang ......................................................................... 471, 489, 567 

Bao, Jialing ...........................................................................................312 

Barber, Glen N. ....................................................................................413 

Barber, Jane .............................................................................................33 

Baricordi, Cristina ........................................................................ 28, 63 

Barish, Michael ...................................................................................648 

Barnard, Alun R. ................................................................................229 

Barnes, Josephine L. .........................................................................434 

Barnes, Justin A. .................................................................................560 

Barrera-L, Araceli ..............................................................................534 

Barrera-Lopez, Araceli ..........................................................246, 564 

Barrett, J. ..................................................................................................15 

Barry, Mary ..........................................................................................583 

Barry, Michael A. ................................................. 132, 481, 583, 748 

Bartel, Melissa A. .................................................................................... 3 

140 



Bartel, Ronnda L. ...............................................................................371 

Bartholomae, Cynthia C. .......................................37, 62, 210, 233, 

.......................................................................... 279, 518, 666, 674, 678 

Bartido, Shirley ...................................................................50, 61, 506 

Bartlett, Jeffrey S. .................................................................................48 

Bartolaccini, Sara ................................................................... 441, 667 

Bärtsch, Marc-Andrea .....................................................................639 

Baruchel, André ....................................................................................62 

Barve, Minal ......................................199, 273, 402, 503, 624, 625 

Barzel, Adi ............................................................................................343 

Bashkirova, Lisa .................................................................................277 

Basil, Maria C. .....................................................................................623 

Basner-Tschakarjan, Etiena ................................................110, 224 

Batshaw, Mark L. ...............................................................................128 

Baum, Bruce J. .....................................................................................743 

Baum, Christopher ........................................37, 66, 313, 320, 425 

Baumgart, J. .........................................................................................694 

Baumhover, Nick J. ...............................................................................41 

Baylink, David J. .................................................................................468 

Beadlescomb, Patricia M. ....................................................... 36, 377 

Bear, Adham S. ....................................................................................269 

Beard, Brian .........................................................................................673 

Beard, Brian C. .................................................................. 25, 311, 421 

Beard, Rachel E. .................................................................................716 

Beaton, Brendan ...................................................................................24 

Beattie, Stuart .....................................................................................274 

Beckman, Sarah .................................................................................611 

Bedell, Cynthia .......................................... 273, 351, 479, 503, 624 

Bee, Youn-Shen ...................................................................................740 

Beggs, Alan H. ............................................................................. 33, 606 

Beggs, Alan ..............................................................................................90 

Begum, Laila ................................................................................ 89, 257 

Behrens, Marshall .............................................................................652 

Beitsch, Peter ................................................................. 199, 624, 625 

Belay, Eyayu ...............................................................................419, 423 

Belbellaa, Brahim ..............................................................................278 

Bell, Jason B. ..............................................................................102, 447 

Bell, John C. ..................................................................................... 19, 22 

Bell, Peter ...................................................................................108, 444 

Bellinger, Dwight A. ..........................................................................733 

Bellinger, Dwight ...............................................................................667 

Bellio, Laura.........................................................................................420 

Belliveau, N. .........................................................................................708 

Bello, Alexander .................................................................................379 

Belur, Lalitha .......................................................................................364 

Bemelmans, Alexis-Pierre .............................................................225 

Bender, Christian ...............................................................................516 

Benedetti, Eric ....................................................................................581 

Benedicenti, Fabrizio .................................64, 233, 309, 517, 666 

Bennet, Alan ...........................................................................................59 

Bennett, Alan ......................................................................................508 

Bennett, Alan D. .................................................................................298 

Bennett, Jean .......................................53, 222, 224, 227, 231, 330 

Bennicelli, Jeannette L. .........................................................224, 227 

Benson, Janet ......................................................................................106 

Berger, Adeline ...................................................................................225 

Berger, Mitchel ...................................................................................703 

Bergeron, E. .........................................................................................694 

Berkley, Brown ...................................................................................307 

Bernal, Yvette ......................................................................................506 

Bernt, William ....................................................................................264 

Berry, Charles .....................................................................................310 

Bertolotti, Roger ......................................................................700, 741 

Betensky, Rebecca A.........................................................................283 

Beyer, Ines .................................................................................... 13, 201 

Beyers, R. J. ..............................................................................................40 

Bhakta, Vipul .......................................................................................561 

Bhargava, Ragini ......................................................................497, 571 

Bian, Tao ................................................................................................449 

Bianchi, Frayne...................................................................................508 

Biasco, Luca ................................................................28, 63, 309, 517 

Bienemann, Alison S. .......................................................................158 

Bifi, Alessandra .......................................................................309, 517 

Binder-Scholl, Gwen .........................................................................508 

Binder-Scholl, Gwendolyn K. ................................................ 59, 298 

Bing, Wang ...........................................................................................614 

Binley, Katie ................................................................................. 56, 232 

Bird, Andrew .......................................................................................618 

Birkett, Ashley .......................................................................................45 

Bishop, Nina ........................................................................................387 

Bisset, Darren R. ................................................................................450 

Bissig, Karl-Dimiter ..........................................................................454 


141


Biswas, Arijit .............................................................................363, 580 

Bjornson, Christopher R. ...............................................................174 

Black, Aaron D. ...................................................................................227 

Black, Aaron H. ...................................................................................224 

Blake, Derek J. .....................................................................................296 

Blanche, Stéphane ................................................................................62 

Blanche, Stephane .............................................................................306 

Blau, C. Anthony.......................................................................419, 423 

Blau, Helen M. .....................................................................................255 

Blazar, Bruce R. ............................................................. 270, 584, 738 

Block, Matthew .....................................................................................17 

Blondeau, Johanna ............................................................................306 

Bloom, Matthew D. ...........................................................................332 

Blumenkrnz, Mark S. ..........................................................................54 

Blundell, Michael ...............................................................................678 

Bobbin, Maggie L. ..............................................................................454 

Bochner, Bernard H. ............................................................................70 

Boerner, Kathleen ...................................................................514, 725 

Bogan, Brooke ....................................................................................522 

Bogan, Dan ..............................................................................................92 

Bogan, Janet ................................................................................. 92, 391 

Bogdanov, R. ........................................................................................724 

Boisgerault, Nicolas ...............................................................395, 652 

Boissel, Sandrine ...............................................................................318 

Bole-Richard, Elodie ........................................................................399 

Bollino, Dominique .................................................................600, 636 

Bolyard, Chelsea M. ................................................................409, 747 

Bonato, Riccardo ...............................................................................433 

Bond, Wesley S. ..................................................................................131 

Bondanza, Attilio ...............................................................................197 

Bonifacino, Aylin ........................................................................ 30, 428 

Bonini, Chiara ................................................................... 28, 197, 504 

Bonnin, Delphine ..................................................................................52 

Bonten, Erik .........................................................................................702 

Bordignon, Claudio .................................................................197, 504 

Borel, Florie ...............................................................................211, 603 

Borelli, Primavera .............................................................................519 

Borg, Christophe ................................................................................399 

Börner, Kathleen ................................................................................123 

Boros, Gábor ........................................................................................147 

Borquez-Ojeda, Oriana ...................................................................506 

Borretta, Lisa ......................................................................................115 

Bortolotti, Francesca ............................................................... 95, 368 

Borzone, Roberta ..............................................................................362 

Boscolo, Francesca ...........................................................................465 

Bosnakovski, Darko ................................................................168, 169 

Bosque, Doyle ........................................................................................10 

Bossi, Giovanna ..................................................................................508 

Bossow, Sascha .............................................................. 405, 554, 639 

Botta, Salvatore ..................................................................................539 

Bouchier-Hayes, Lisa .......................................................................684 

Bougnères, Pierre ................................................................................62 

Bouinta, Asimina ..................................................................................27 

Boulad, Farid ..........................................................................................61 

Boulis, Nicholas M. ...........................................................................287 

Bourdel, Alix ........................................................................................731 

Bourg-Alibert, Nathalie ..................................................................615 

Boutin Fontaine, Marjorie ................................................................52 

Boyd, Maureen P. ..................................................................................48 

Boye, Sanford E. .................................................................................596 

Boye, Sanford L. ............................................................... 55, 115, 223 

Boye, Shannon E. ....................................................................... 55, 223 

Bradbury, A. M. ......................................................................................40 

Bradbury, Allison M. .............................................36, 377, 590, 592 

Braga, Luca ..............................................................................................95 

Brambilla, Aurora ..............................................................................504 

Brantly, Mark .......................................................................................545 

Braun, Stephen E. ..............................................................................195 

Breakeield, Xandra O. ...................................... 103, 286, 304, 751 

Bredius, Robbert G. M. ....................................................................313 

Breitbach, Caroline H. ........................................................................22 

Breitbach, Caroline J. ..........................................................................19 

Bremer, William G. ...............................................................................91 

Brendel, Christian ................................................................................66 

Brendler, Charles ...............................................................................555 

Brenner, Gary J. ..................................................................................286 

Brenner, Malcolm K. .........................14, 299, 300, 398, 622, 684 

Brennig, Sebastian ..................................................................320, 429 

Brentjens, Renier.....................................................................506, 651 

Breton, Louis R. .....................................................................................48 

Brewer, Joanna ........................................................................... 59, 508 

Brewer, Joanna E. ..............................................................................298 

142 



Bridges, Charles R. ............................................................................374 

Bringas, John .................................................................. 288, 594, 599 

Broderick, Kate E. ...................................................................153, 341 

Brooks, Elizabeth J. ...........................................................................276 

Brooks, Susan .....................................................................................295 

Broome, Ted ...........................................................................................88 

Brough, Douglas E. ............................................................................737 

Brown, Ami S..........................................................................................45 

Brown, Christine ...............................................................................648 

Brown, Harrison C. ...........................................................................663 

Brown, Jillian R. .................................................................................365 

Brown, Jr, Robert H. .............................................................................93 

Brown, Kevin ..........................................................................................51 

Brown, Matthew T. ..................................................................292, 491 

Brown, Patricia...................................................................................351 

Brown, Rachel E. ................................................................................434 

Brown, Robert ..........................................................................388, 603 

Brown, Ryan ........................................................................................721 

Brownlie, Joe .......................................................................................185 

Brugman, Martijn H. ..............................................................319, 425 

Brunetti-Pierri, Nicola ..................................... 353, 362, 499, 553 

Brunger, Jonathan M. .......................................................................241 

Brunhoeber, Elaine ...........................................................................461 

Brunicardi, F. C. ..................................................................................659 

Brunicardi, F. Charles ................................................. 272, 624, 625 

Brunson, Brandon L. ........................................................................590 

Brusko, Todd M. .................................................................................109 

Bu, Le ......................................................................................................750 

Buckvold, Megan P. ...........................................................................495 

Bueren, Juan Antonio ......................................................................678 

Buggio, Maurizio ...............................................................................438 

Bui, Jacquelin H. .................................................................................107 

Buj-Bello, Anna ...............................................33, 90, 240, 605, 606 

Bundschuh, Ralf .................................................................................412 

Bunnell, Bruce A. ...............................................................................195 

Burcus, Niculina ....................................................................................99 

Burke, Bryan P. ......................................................................................48 

Burke, James ...........................................................................................19 

Burkin, Dean J. ....................................................................................175 

Burnett, Ryan ......................................................................................703 

Burrascano, Cindy .............................................................................703 

Burtner, Christopher R. ........................................................311, 421 

Bushman, Frederic .................................................................306, 310 

Butts, Tiffany .......................................................................................443 

Byrne, Barry J............................................ 57, 60, 90,376, 446, 617 

Byrne, Leah C. .......................................................................................... 5 

Byrnes, Andrew P. .............................................................................133 

Byun, Jung-Ick ............................................................... 143, 598, 602 

Byung-Rok, Do ....................................................................................699 

Caccavelli, Laure ......................................................................306, 310 

Cagnina, Shannon..............................................................................351 

Cai, Ying .................................................................................................263 

Calabria, Andrea .......................................................28, 63, 309, 517 

Calcedo, Roberto ...............................................................................360 

Calos, Michele P. .................................................................................174 

Camadro, Jean-Michel .....................................................................612 

Camba-Colon, Joanna .........................................................................48 

Cameron, Brian ..................................................................................508 

Camisa, Barbara .................................................................................197 

Campell-Thompson, Martha .........................................................721 

Campo, John ........................................................................................184 

Campos, Maria ....................................................................................709 

Cancelas, Jose ......................................................................................426 

Candotti, Fabio .........................................................................307, 437 

Cannizzo, Stefania E. ..............................................................315, 469 

Cannon, Paula M. .......................................................... 183, 184, 487 

Cantore, Alessio .................................................. 441, 496, 666, 667 

Cao, Chunxia ........................................................................................335 

Cao, Hua .............................................................................. 13, 201, 303 

Cao, Hua ................................................................................................679 

Cao, Huibi .............................................................................................433 

Cao, Ou ...................................................................................................190 

Cao, Phuong .........................................................................................248 

Cappellini, Maria Domenica .........................................................420 

Capracotta, Sonja ..............................................................................264 

Carbajo, Rosangela .................................................................152, 620 

Carbonaro, Denise A. .......................................................................307 

Carlier, Pierre ......................................................................................734 

Carlisle, Holly ............................................................................339, 550 

Carlson, Daniel F. ...............................................................................738 

Carnes, Aaron E. .................................................................................263 

Carnes, Aaron .....................................................................................261 


143


Carney, Gael G. ....................................................................................672 

Carroll, Maria V......................................................................................48 

Carruthers, K. ......................................................................................695 

Cartier, Nathalie ......................................................................... 62, 279 

Caruana, Ignazio ................................................................................392 

Cassinerio, Elena ...............................................................................420 

Castaldo, Sigismondo ......................................................................353 

Castello, Raffaele ...............................................................................362 

Castle, Michael J. ................................................................................116 

Castleman, William ..........................................................................721 

Castleton, Anna Z. .............................................................................411 

Castleton, Anna .....................................................................................24 

Casucci, Monica ..................................................................................197 

Caswell, Debbie ..................................................................................436 

Cataldi, Marcela P. .............................................................................125 

Catalgol, Betul .....................................................................................697 

Cataliotti, Alessandro ......................................................................746 

Catani, Joao Paulo P. .........................................................................630 

Cathomen, Toni ..................................................................................539 

Cattaneo, Roberto .............................................................................415 

Cavazzana-Calvo, Marina .....62, 279, 306, 310, 313, 661, 662 

Cederbaum, Stephen D. ..................................................................497 

Celis, Esteban ......................................................................................395 

Certoux, Jean-Marie .........................................................................399 

Cerullo, Vincenzo ....................................................................501, 607 

Cesana, Daniela .......................................................................... 64, 233 

Cesani, Martina ........................................................................309, 517 

Chérel, Yan ...........................................................................................111 

Chai, Jing ...............................................................................................476 

Chai, Kuan .............................................................................................216 

Chalberg, Thomas W. ..........................................................................54 

Chamberlain, Jeffrey S. ....................................................................543 

Chamberlain, Joel R. .........................................................................450 

Chambers, Christopher B. ........................................... 26, 200, 235 

Champlin, Richard E. .................................10, 621, 626, 649, 653 

Chan, Jerry ....................................................................... 363, 430, 580 

Chan, Jerry K. Y. ..................................................................................254 

Chan, T. ......................................................................................................15 

Chandler, Randy J. .............................................................................113 

Chandra, Manasa ...............................................................................542 

Chandrasekaran, Devikha..............................................................421 

Chang, Esther H. ................................................................................273 

Chang, Susan M. .................................................................................676 

Chang, Wen-Teng ...............................................................................723 

Chan-Seng, Delphine ........................................................................157 

Chapdelaine, Pierre ..........................................................................389 

Chapman, Michael S. ........................................................................336 

Chard, Louisa S. ..................................................................................507 

Charrier, Sabine .................................................................................306 

Charton, Karine ........................................................................610, 615 

Chatterjee, Saswati ...........................................................................542 

Chaudhuri, Aadel A. ..........................................................................712 

Chaurasiya, Shyambabu .................................................................403 

Chavez, Christopher L. ....................................................................174 

Chen, Alvin ...........................................................................................589 

Chen, Dalin ...........................................................................................474 

Chen, Hsing-Yu ....................................................................................348 

Chen, Jeremy. J.W. ..............................................................................348 

Chen, Li ........................................................................................464, 570 

Chen, Liping .........................................................................................665 

Chen, Mong-Jen ..................................................................................545 

Chen, Pao-Rong ..................................................................................723 

Chen, Qiumei .......................................................................................369 

Chen, S....................................................................................................708 

Chen, Stacey .........................................................................................373 

Chen, Thomas .....................................................................................703 

Chen, Vincent C. .................................................................................474 

Chen, Vincent ......................................................................................476 

Chen, Weilin .........................................................................................751 

Chen, Wenyi .........................................................................................474 

Chen, Xinglu .........................................................................................221 

Chen, Yifeng ...............................................................................110, 498 

Chen, Yvonne Y. ..................................................................................744 

Chen, Zhi-Ying .....................................................................................270 

Cheng, Seng H. .................................................................. 34, 282, 453 

Chen-Wichmann, Linping .................................................................66 

Cherel, Yan ...........................................................................................734 

Cherqui, Stephanie ...................................................... 493, 494, 546 

Cheung, Aaron ....................................................................................593 

Chi, Yudan .............................................................................................562 

Chiang, Andy .......................................................................................665 

Chiara, Bovolenta ..............................................................................520 

144 



Chicoine, Louis G. .................................................................................91 

Chien, Yin-Hsiu ........................................................................... 60, 376 

Chikovani, T .........................................................................................682 

Childers, Martin ......................................................................... 90, 391 

Childers, Martin K. ................................................33, 473, 606, 617 

Chiocca, E. Antonio .................................................................676, 690 

Chiodo, Vince A. .................................................................................596 

Chiodo, Vince ......................................................................................545 

Chion-Sotinel, Isabelle ....................................................................401 

Chiou, Shih-Hwa ................................................................................720 

Chiuchiolo, Maria J. ................................................................... 11, 106 

Chiyo, Tomoko ............................................................... 120, 176, 608 

Cho, Jung Jong .....................................................................................565 

Cho, Mong ........................................................................................ 19, 22 

Cho, Soo Kyung ........................................................................... 42, 163 

Chodon, Thinle ...................................................................................302 

Choi, Boyoung .....................................................................................238 

Choi, E. ...................................................................................................695 

Choi, Eun-Young .......................................................................383, 586 

Choi, H. ...................................................................................................708 

Choi, Il-Kyu ...................................................................... 129, 679, 689 

Choi, Ji Ye ..............................................................................................602 

Choi, Joung-Woo ......................................................................556, 689 

Choi, Ju Hyun .......................................................................................375 

Choi, Kyoung Baek ............................................................................138 

Choi, Min Jeong ..................................................................................375 

Choi, Uimook ............................................................................... 82, 308 

Choolani, Mahesh ......................................................... 363, 430, 580 

Chorny, Michael............................................................. 144, 366, 372 

Chou, Alice Y. .......................................................................................115 

Chou, Szu-Ting .......................................................................................44 

Choudhury, Sourav ................................................................................ 1 

Christiansen, Lauryn ............................................................... 37, 251 

Christiansen, Lauryn M. .................................................................258 

Christiansen-Weber, Trudy .................................................137, 470 

Chu, Jun .................................................................................................255 

Chu, Kirk ................................................................................7, 191, 681 

Chu, Kon ........................................................................... 143, 598, 602 

Chu, Tiffany ................................................................................137, 470 

Chu, Yongchao ....................................................................................561 

Chuah, Marinee K. .................................................................................. 8 

Chuah, Marinee ............................................................. 355, 496, 667 

Chuah, Marinne ..................................................................................354 

Chuang, Chen-Hua ............................................................................436 

Chung, Daniel C. .......................................................................160, 227 

Chung, Daniel ......................................................................................224 

Chung, Janet .........................................................................................668 

Chung, Su I. ..........................................................................................513 

Ciceri, Fabio ...............................................................................420, 504 

Cicieri, Fabio ........................................................................................197 

Cieri, Nicoletta ............................................................................ 28, 504 

Cingoz, Ahmet .....................................................................................697 

Ciriello, Giovanni ...............................................................................728 

Claesson-Welsh, Lena ......................................................................631 

Clark, K. R. ...............................................................................................91 

Clark, K. Reed ................................................................. 171, 182, 291 

Clark, Katherine A. ............................................................................366 

Clark, Randall ......................................................................................284 

Claudiani, Pamela ....................................................................359, 360 

Claudio, Bordignon ...........................................................................520 

Clayton, Nicholas P. ...........................................................................453 

Cleaver, Brian .........................................................................................55 

Clemens, Paula R. .................................................................................94 

Clemente, Nathalie ..............................................................................55 

Clevers, Hans ..........................................................................................85 

Cliby, William .........................................................................................17 

Clifford, Brian T. .................................................................................369 

Cloer, Caryn ..........................................................................................451 

Cloughesy, Timothy F. ......................................................................676 

Coates, Allan L. ...................................................................................433 

Cocchiarella, Fabienne ....................................................................347 

Coffey, Matt .................................................................................. 18, 652 

Cohen, Justus B. ............................................................. 416, 478, 566 

Cohen, Justus .......................................................................................285 

Cohen-Haguenauer, Odile .................................................................64 

Colahan, Patrick ....................................................................................88 

Cole-Strauss, Allyson .......................................................................386 

Collier, T. H. ..........................................................................................381 

Collier, Timothy J. ..............................................................................386 

Collins, Christopher J. ......................................................................745 

Collins, Shelley ......................................................................................57 

Colosi, Peter .........................................................................................709 


145


Colunga, Aric .......................................................................................636 

Conlon, Thomas J.......................................................................... 55, 57 

Conlon, Thomas .......................................................................545, 721 

Connolly, Jeanne M. ..........................................................................366 

Constable, Ian J. .....................................................................................54 

Constantinou, Varnavas .....................................................................27 

Contag, Christopher H. ....................................................................255 

Cook, P. ......................................................................................................53 

Cooper, Laurence J. N. .........................................10, 198, 202, 393, 

......................................................431, 502, 621, 626, 649, 650, 653 

Cooper, Mark J. ...................................................................................344 

Corbel, Stephane Y. ...........................................................................255 

Cornetta, Kenneth .............................................................................703 

Corrales, Ricardo A. ..........................................................................366 

Cortado, Ruth ......................................................................................530 

Cossette, Travis L. ................................................................................55 

Cossette, Travis ..................................................................................721 

Cost, Gregory J. ...................................................................................568 

Cotrim, Ana P. ......................................................................................743 

Cotugno, Gabriella ..................................................................359, 360 

Coulombe, Zoé ....................................................................................389 

Courne, Celine ....................................................................................674 

Courtney, Amy .......................................................................................12 

Couture, Larry ..........................................................................474, 476 

Cox, N. R....................................................................................................40 

Cox, Nancy R. .............................................................................590, 592 

Cradick, Thomas J. ............................................. 324, 471, 489, 567 

Cram, Sara A. .......................................................................................589 

Crane, Ana M. ......................................................................................107 

Cranill, Palula J. .................................................................................255 

Crawford, Brett E. ..............................................................................365 

Crawford, Gregory E. .......................................................................241 

Cripe, Timothy P. ...............................................................................412 

Cristea, Sandra ...................................................................................568 

Crommelin, Daan ...............................................................................707 

Crommentuijn, Matheus H. W. .....................................................540 

Crosby, Catherine M. ........................................................................132 

Cross, Guillem .....................................................................................306 

Crossland, Denise L. .........................................................................198 

Crudele, Julie M. .................................................................................498 

Crystal, Ronald G. ..............................11, 106, 278, 373, 589, 727 

Cullis, P. ..................................................................................................708 

Cuneo, Nicasio ....................................................................................635 

Curiel, David T. .................................127, 246, 438, 558, 560, 564 

Curinga, Gabrielle ................................................................................32 

Curinga, Gabrielle M. .......................................................................673 

Curren, Elizabeth...............................................................................387 

Curtis, Sarah ........................................................................................276 

Cutler, Benjamin ................................................................................108 

Cutrera, Jeffry .....................................................................................139 

Cutz, Ernest..........................................................................................433 

Cyckowski, L. ..........................................................................................53 

Czarnecki, Kevin ................................................................................429 

Czech, Michael P. ...................................................................................96 

da Silva, Flavia H. ...............................................................................582 

Daenthanasanmak, Anusara............................................................47 

Daguindau, Etienne ..........................................................................399 

Dahlmann, Julia ..................................................................................320 

Dai, Jihong ............................................................................................681 

Dai, Mei .......................................................................................... 38, 248 

Dai, Mushui .............................................................................................72 

Dai, Yifan ...............................................................................................281 

Dal Ferro, Matteo..................................................................................95 

Dal-Cortivo, Liliane ...........................................................................306 

Dalkara, Deniz ......................................................................................... 5 

Dalle, Jean-Hughes ...............................................................................62 

Dandapat, Abhijit ..............................................................................169 

Dane, Allison P. ...................................................................................356 

Daneshmand, Manijeh .......................................................................19 

Dang, Wei ....................................................................................474, 476 

D’Angelo, Armando .................................................................496, 667 

Daniele, Nathalie ......................................................33, 90, 605, 610 

D”Aria, Stefania ...................................................................................362 

Danos, Olivier .....................................................................................731 

Danuta, Jarocha ..................................................................................714 

Dar, Gulam H. ......................................................................................156 

Darabi, Radbod ........................................................................168, 169 

Darvish, Babak .........................................................................152, 620 

Darvish, Daniel .........................................................................152, 620 

Dashkoff, Jonathan ...........................................................................283 

Dassie, Justin P. ...................................................................................509 

Dator, Dante .........................................................................................654 

146 



Daukandt, Marc ..................................................................................261 

Davidson, Beverly L. .........................................................................283 

Davidson, Michael W. .......................................................................255 

Davidson, Robert H. .........................................................................110 

Davidson, Robert J. ...........................................................................488 

Davila, Alejandra ...............................................................................307 

Davila, Marco L. ..................................................................................506 

Davis, Brian R. ....................................................................................107 

Davis, Carol ..........................................................................................295 

Davis, Daniel ...........................................................................................24 

Davis, Gregory D. ...............................................................................574 

Davydova, Julia .................................................... 408, 414, 705, 742 

Dawson, Brian ....................................................................................607 

Dawson, Margaret ................................................................................10 

d’Azzo, Alessandra ............................................................................702 

de Alencastro, Gustavo ...................................................................356 

de Andreade Pereira, Bruna .........................................................736 

De Bleser, Pieter .................................................................................355 

de Boer, Helen ...........................................................................275, 661 

De Deyn, Peter ....................................................................................497 

de Fougerolles, Antonin .................................................................463 

de Leeuw, Charles N. ........................................................................115 

de Mollerat du Jeu, Xavier .............................................................706 

de Moraes, Jane Z. .............................................................................658 

de Oliveira, Satiro ..............................................................................307 

De Palma, Michele .............................................................................301 

de Prisco, Nicola ................................................................................539 

De Ravin, Suk See ..............................................................................308 

De Saint Basile, Geneviève ............................................................662 

De Silva Feelixge, Harshana ..........................................................187 

De, Bishnu P. ................................................................................ 11, 589 

Dean, David A. ........................................................................................43 

Deconinck, Eric ..................................................................................399 

Deeg, H. Joachim ................................................................................525 

DeFeo, Anthony P. ...................................................................584, 738 

DeFrees, Kyle .......................................................................................745 

Degele, Meghan ..................................................................................273 

Deichmann, Annette ........................................................................210 

Del Vecchio, Michele ........................................................................394 

Delaney, Colleen .................................................................................249 

Delaney, Kyle .......................................................................................524 

Delebecque, Frederic ..........................................................................48 

Delhove, Juliette M. ...........................................................................435 

Della Valle, Patrizia .................................................................496, 667 

DeMaster, Amanda ............................................................................276 

Demonte, Marina ...............................................................................635 

Denapoli, Priscila M. A. .........................................................265, 658 

Denard, Jerome ..................................................................................445 

Denaro, Maria ............................................................................. 37, 674 

Denes, Bela ...........................................................................................507 

Deng, Wen-Tao .......................................................................................55 

Deniger, Drew C. ................................................................................502 

Denney, T. S. ............................................................................................40 

Denning, Gabriela .............................................................................528 

Dennis, James E. ....................................................................................96 

DeRavin, Suk See ..................................................................................82 

Derniame, Sophie ..............................................................................401 

Deschamps, Jack-Yves ......................................................................111 

Deschamps, Marina ..........................................................................399 

Desrosiers, Mélissa ...........................................................................225 

Devaux, Marie .....................................................................................111 

Dey, Aditi ....................................................................................... 24, 411 

Deyle, David R. ....................................................................................266 

Dhamne, Chetan ................................................................................431 

Di Matteo, Mario ......................................................................355, 496 

Di Serio, Clelia ........................................................................................63 

Di Stasi, Antonio ................................................................................684 

Di Tomaso, Tiziano ...........................................................................486 

Di, Wei-Li ..............................................................................................578 

Diago, Oscar .........................................................................................703 

Diamond, Scott L. ..............................................................................374 

Diao, Yong .............................................................................................154 

Diaz, Rosa .................................................................18, 395, 505, 652 

Dichek, David ......................................................................................559 

Dickey, David D. ..................................................................................327 

Dickson, Georges ...............................................................................734 

Dickson, Patricia I. ............................................................................586 

Dietz, Wynette M. ..............................................................................270 

Dighe, Niraja ........................................................................................430 

DiGiusto, David L. ..............................................................................668 

Dillberger, Alexa.................................................................................137 

Ding, W. ..................................................................................................694 


147


Dionisio, Francesca .............................................................................63 

Discher, Dennis E. ..............................................................................523 

Disis, Nora ...............................................................................................13 

Dismuke, David J. ..................................................................................54 

Dissen, Gregory A. ................................................................................78 

Djuric, Ugljesa .....................................................................................593 

Do, Byung-Rok ....................................................................................722 

Dobbins, George C. ............................................................................127 

Doerler, Phillip A. .............................................................................446 

Doering, Christopher B. ................................................ 31, 528, 663 

Doering, Jon ............................................................................................33 

Doglioni, Claudio .....................................................................233, 666 

Doherty, Joseph E. .............................................................................269 

Doki, Yuichiro .....................................................................................749 

Domingo Musibay, Evidio ...................................................... 23, 641 

Donahue, Robert .................................................................29, 30, 428 

Donahue, Robert E. ..............................................................................65 

Dondero, Richard ..............................................................................214 

Dong, Biao ............................................................................................681 

Donnelly, Eleanor M. ........................................................................287 

Donnelly, Oliver ......................................................18, 395, 505, 652 

Donsante, Anthony .................................................................383, 586 

Dore, Franca ........................................................................................368 

Dorer, Dominik E. .................................................................................21 

Doria, Monica ......................................................................................340 

Doronin, Konstantin ........................................................................477 

Dorrell, Craig ............................................................................... 85, 581 

Dorward, Heidi ...................................................................................437 

Dotti, Gianpietro ............................................. 12, 14, 49, 197, 299, 

.......................................................................... 300, 305, 392, 394, 684 

Dou, Chunqing ....................................................................................616 

Dow, David ...........................................................................................309 

Dowdy, Sean ...........................................................................................17 

Doyal, Mark F. ......................................................................................285 

Doyon, Yannick ...................................................................................488 

Dresch, Christiane .............................................................................736 

Driessen, Gertjan J. A. ......................................................................313 

Drivas, Theodore G. ................................................................222, 231 

Dropulic, Boro ....................................................................................495 

Du, Junkai .............................................................................................616 

Du, Kai ....................................................................................................433 

Du, Liang ...............................................................................................559 

Duan, Cathleen ...................................................................................433 

Duan, Dongsheng ..................................................98, 166, 175, 613 

Duan, Yuyou .........................................................................................468 

Dubois, Kara ...........................................................................................19 

Dubreil, Laurence ...................................................................... 76, 111 

Duchateau, Philippe .........................................................................731 

Dufour, Brett ........................................................................................284 

Dukes, Joseph ......................................................................................508 

Dulatova, Galina .................................................................................619 

Dumitriu, Bogden .................................................................................65 

Dunbar, Cynthia E. ....................................................................... 29, 65 

Duncan, F. Jason ......................................................................... 39, 597 

Duncker, Dirk J. ...................................................................................275 

Duong, Jennifer .....................................................................................46 

Dupont, Jean Baptiste .........................................................................76 

Duros, Caroline .....................................................................................64 

Duskey, Jason ......................................................................................155 

Dusting, Gregory J. ..................................................................230, 740 

Dutilleul, Maeva .................................................................................111 

Düzgünes, Nejat .................................................................................576 

Dyka, Frank M. ..........................................................................115, 223 

Dyke, Jonathan P. ..................................................................................11 

Earley, Lauriel F. ....................................................................................72 

Earley, Lauriel .....................................................................................259 

Eastlund, Erik R. ................................................................................243 

Ebihara, Yasuhiro .................................................................................86 

Edelman, Aleksander .......................................................................731 

Edelman, Gerald ................................................................................273 

Egerer, Lisa ...........................................................................................195 

Egger, Erica ..........................................................................................530 

Eguchi, Akiko ......................................................................................706 

Ehrhardt, Anja ....................................................................................557 

Eiden, Maribeth V. .............................................................................524 

El Khoury, Joseph ..............................................................................304 

Elahi, Seyyed Mehdy ........................................................................135 

Elamouri, Salim S. .............................................................................248 

El-Amouri, Salim S. ..............................................................................38 

El-Attar, Laila M..................................................................................185 

Elazar, Menashe .................................................................................191 

Elder, James .........................................................................................676 

148 



Eleftheriadou, Ioanna .........................................................................67 

Elizabeth, Garabedian .....................................................................307 

Elkouby, Liron .....................................................................................110 

Ellis, James .................................................................................322, 593 

Ellis, Scott ..................................................................................... 56, 232 

Ellison, Stuart M. ..................................................................................67 

Elnaggar, Atif .......................................................................................208 

Emara, M.M. .........................................................................................682 

Embry, Addie .......................................................................................505 

Emery, David W. .......................................................................419, 423 

Emery, Lyndsey A. ................................................................................59 

Emri, Eszter .........................................................................................147 

Emri, Gabriella ...................................................................................147 

Emrick, Todd .......................................................................................157 

Endo-Takahashi, Yoko......................................................................142 

Engeland, Christine E. .....................................................................405 

Engelhardt, John F. ............................................................................535 

Engelhardt, Sarah ...................................................................... 21, 554 

Engler, Tatjana ..........................................................................126, 484 

Epperly, Michael W. ..........................................................................239 

Eren, Fatih ............................................................................................452 

Erez, Ayelet ..........................................................................................607 

Erger, Kirsten E. ....................................................................................55 

Erger, Kirsten ......................................................................................545 

Erger, Kirsten ......................................................................................721 

Ericson, Mark E. ....................................................................................41 

Escamilla-Powers, Julie ..................................................................454 

Escobar, Giulia ..........................................................................301, 486 

Escolar, Maria ........................................................................................62 

Esko, Jeffrey D. ....................................................................................448 

Esmaeili Shandiz, Alaleh ................................................................472 

Esmaeili-Shandiz, Alaleh ................................................................575 

Espenlaub, Sigrid ..............................................................................134 

Espinoza, Luis A. ................................................................................217 

Essand, Magnus .................................................................................631 

Estévez, Molina ..................................................................................678 

Eto, Yoshikatsu .........................................................................365, 585 

Eto, Yoshikatsu ...................................................................................753 

Evans, Molly ................................................................................. 30, 428 

Evens, Hanneke ............................................................................. 8, 496 

Excoffon, Katherine J. ......................................................................327 

Excoffon, Katherine J. D. A. ............................................................267 

Exline, Colin .........................................................................................184 

Exline, Colin M. ...................................................................................487 

Eyquem, Justin ....................................................................................401 

Fabre, Peggy ........................................................................................225 

Facher, Evan .........................................................................................500 

Faella, Armida .....................................................................................110 

Faella, Arminda ..................................................................................733 

Fairbanks, Carolyn A. .......................................................................364 

Falcione, Antonella ..............................................................................95 

Falcone, Laura ....................................................................................197 

Falk, Darin ............................................................................................739 

Fan, Luping ................................................................................101, 140 

Fang, Jin ....................................................................................................23 

Farag, Jacqueline ..................................................................................92 

Farah, Benjamin .................................................................................618 

Fargnoli, Anthony S. .........................................................................374 

Farin, Alicia M. ....................................................................................241 

Fariss, Robert ......................................................................................709 

Farmer, Andrew .................................................................................532 

Farrugio, Alfonso P. ...........................................................................174 

Faure-Kumar, Emmanuelle ...........................................................522 

Faust, Susan ...............................................................................108, 444 

Federici, Thais ....................................................................................287 

Federoff, Howard J. ...........................................................................457 

Federspiel, Mark J. ............................................................................413 

Federspiel, Mark ...................................................................................17 

Fedsteinand, Ariel E. ........................................................................706 

Fehling, Hans Jörg .............................................................................445 

Feldman, Steve A. ..............................................................................716 

Felsburg, Peter J. ................................................................................311 

Fender, Pascal .....................................................................................552 

Fender, Pascal .....................................................................................729 

Feng, Nathan .......................................................................................656 

Feng, Yi ..................................................................................................533 

Fergus, Jeffrey .....................................................................................244 

Ferla, Rita ....................................................................................359, 360 

Fernandez-Ulibarri, Ines ...................................................................21 

Ferrand, Christophe .........................................................................399 

Ferrara, Antonella .............................................................................340 

Ferrari, Giuliana .................................................................................420 


149


Ferraro, Bernadette .................................................................. 45, 397 

Ferreira, Jennifer ................................................................1, 384, 595 

Ferro, Leda ..............................................................................................61 

Ferrone, Soldano ...............................................................................394 

Festag, Marvin .......................................................................................81 

Fewell, Jason G. ...................................................................................461 

Fibbe, Willem E. .................................................................................319 

Fielding, Adele K. ....................................................................... 24, 411 

Fife, Jeremy ..........................................................................................162 

Figliola, Matthew J. ..............................................................................10 

Figueiredo, Constanca ........................................................................47 

Filareto, Antonio ................................................................................168 

Findlay, Andrew R. ............................................................................180 

Fine, Bruce ...........................................................................................503 

Fine, Eli J. ......................................................................... 324, 489, 567 

Finegold, Milton .......................................................................316, 466 

Fineman, Jeffrey R. ............................................................................369 

Finer, Michell .......................................................................................674 

Finer, Mitch ................................................................................260, 262 

Finer, Mitchell ............................................................................. 37, 251 

Finkelstein, Jeffrey ............................................................................298 

Finlayson, Neil ....................................................................................263 

Finn, Eric ...............................................................................................543 

Finn, Jonathan D. ...............................................................................498 

Finocchiaro, Liliana M. E. ...............................................................632 

Firrito, Claudia ...................................................................................486 

Fischbeck, Kenneth H. .....................................................................378 

Fischer, Alain ...........................................................62, 279, 306, 310 

Fischer, Anne C. ..................................................................................361 

Fischer, Ashley L. ...............................................................................243 

Fischer, David L. .................................................................................386 

Fishbein, Ilia ..............................................................................366, 372 

Fitzgibbons, Timothy P. .....................................................................96 

Flake, Alan W. ............................................................................236, 418 

Flanigan, Kevin M. .............................................................................180 

Flannery, John G. ..................................................................................... 5 

Flatt, Justin W. .....................................................................................477 

Fleming, S. M. ......................................................................................381 

Fleminig, Sheila M. ............................................................................386 

Flotte, Terence R. .............................................4, 211, 326 361, 735 

Fodor, Istvan ..............................................................................507, 561 

Foley, Erin .............................................................................................448 

Foley, Kevin T.......................................................................................619 

Follenzi, Antonia ......................................................................315, 469 

Foltz Greg ..............................................................................................676 

Fong, Mei ...............................................................................................532 

Fontes, Andrew ..................................................................................244 

Forbes, Scott P. ..........................................................................366, 372 

Forget, Marie A. ..................................................................................621 

Forsayeth, John ............................................................. 288, 594, 599 

Fortuno, Lowella V. ...........................................................................382 

Foster, Aaron .......................................................................................269 

Fox, Catherine .....................................................................................664 

Fox, Tara L. ...........................................................................................477 

Fraefel, Cornel ....................................................................................736 

Fragata, Rowell .........................................................................363, 580 

Francis, Sarah......................................................................................214 

Franco-Bourland, Rebecca E. .............................................246, 564 

Frederick, Amy ...................................................................................338 

Freemark, Michael S.........................................................................276 

Frelan, Megan .....................................................................................655 

French, Deborah L.............................................................................224 

French, Martyn ......................................................................................54 

Frenk, Esther Z. .......................................................................... 11, 106 

Frenzke, Marie ....................................................................................415 

Frey, William H. ..................................................................................364 

Freyvert, Yevgeniy ............................................................................568 

Frigault, Matthew J. ..........................................................................623 

Frittoli, Marta ......................................................................................420 

Fritz, Ashley .........................................................................................692 

Fronza, Raffaele .................................................. 204, 210, 274, 518 

Fry, Terry J. ...........................................................................................325 

Fu, Freddie ...................................................................... 281, 290, 614 

Fu, Haiyan ........................................................................... 39, 125, 597 

Fu, Pei-yu ..............................................................................................307 

Fujimoto, Naoko ......................................................................357, 715 

Fujisawa, Masato .....................................................................192, 654 

Fujita, Ryo ................................................................................................80 

Fukuda, Kuniaki .................................................................................643 

Fukuda, Takahiro .....................................................................365, 585 

Fulci, Giulia ..........................................................................................286 

Funato, Kosuke ......................................................................................11 

150 



Furth, Mark E. ........................................................................................33 

Furth, Mark ..........................................................................................473 

Furukawa, Yoichi ..................................................................................86 

Fyles, Thomas M. ...............................................................................755 

Gabriel, Nishanth ....................................................................114, 549 

Gabriel, Richard ............................................................ 518, 678, 738 

Gabrielse, Kari ....................................................................................710 

Gaddameedi, Rajashekhar ...................................................224, 227 

Gadkari, Rupali ...................................................................................536 

Gadkari, Rupali A. ..............................................................................114 

Gadue, Paul ..........................................................................................224 

Gahl, William A. ..................................................................................437 

Galanis, Evanthia ......................................................17, 23, 417, 641 

Galetto, Roman ...................................................................................401 

Galetto, Roman ...................................................................................683 

Galibert, Lionel ......................................................................................52 

Galivo, Feorillo ..........................................................................466, 581 

Gallina, Pierangela ..................................................................233, 666 

Galtseva, I. ............................................................................................724 

Galy, Anne ...................................................................................306, 662 

Gan, Chih Hao V..................................................................................678 

Gangemi, Mariela A. .........................................................................635 

Gannon, Frank ....................................................................................607 

Gannon, Mary......................................................................................587 

Ganser, Arnold .......................................................................................47 

Gao, Chun ..............................................................................................709 

Gao, Ehre ...............................................................................................444 

Gao, Guangping .......................4, 35, 93,128, 326, 328, 384, 512 

Gao, Mingming....................................................................................750 

Gao, Shijuan .........................................................................................194 

Gao, Xueqin ..........................................................................................178 

Gaponova, T..........................................................................................724 

Garcia, Elizabth ..................................................................................648 

Garcia, Luis...........................................................................................225 

Garcia-Figueroa, Jorge ..........................................................152, 620 

Garín, Marina I....................................................................................678 

Garitaonandia, Ibon .........................................................................465 

Garwick-Coppens, Sara ...................................................................295 

Gaudet, Daniel ....................................................................................274 

Gebremedhin, Senait .......................................................................576 

Gee, Adrian P. .............................................................................. 14, 299 

Gee, J. .........................................................................................................53 

Geekiyanage, Hirosha .........................................................................23 

Geguchadze, Ramaz ...............................................................141, 149 

Geiger, Sabine.........................................................................................26 

Geisinger, Jonathan M. .....................................................................174 

Genovese, Pietro ........................................................... 197, 486, 504 

Gentner, Bernhard ..................................................................197, 420 

Georger, Christophe ............................................................................76 

Georgiadis, Christos .........................................................................578 

Gerber, Mark A....................................................................................243 

Gerdemann, Ulrike............................................................................396 

Gernoux, Gwladys .............................................................................111 

Gerry, Andrew ....................................................................................508 

Gerry, Andrew B. ....................................................................... 59, 298 

Gersbach, Charles A. ...........167, 170, 177, 241, 292, 293, 491 

Getz, Matthew .....................................................................................721 

Geurten, Olivier ..................................................................................261 

Geurts, Aron ........................................................................................746 

Ghandeharian, Omid ............................................................................. 8 

Ghashghaei, Troy .................................................................................... 2 

Ghatrora, Rupinder K. .....................................................................434 

Ghisoli, Maurizio......................................................................203, 624 

Ghivizzani, Steve ................................................................................335 

Ghivizzani, Steven ................................................................................88 

Ghoda, Lucy Y. .....................................................................................648 

Gholizadeh, Shervin .........................................................................390 

Giacca, Mauro ............................................................................. 95, 368 

Gian Paolo, Rizzardi .........................................................................520 

Giangrande, Paloma H. ..........................................................509, 511 

Giannini, Caterina .............................................................................417 

Giardina, Patricia ..................................................................................61 

Gicquel, Evelyne .......................................................................610, 615 

Giedlin, Marty ........................................................................................58 

Gilbert, Daniel .....................................................................................514 

Gilbert, Rénald ....................................................................................135 

Gilbert, Shawn ....................................................................................548 

Gilchrist, Megan .................................................................................648 

Giles, April ............................................................................................116 

Gilkes, Janine A. ..................................................................................332 

Gill, Deborah R....................................................................................460 

Gillet-Legrand, Beatrix ....................................................................674 


151


Giordano, Frank .................................................................................518 

Giovani, Michelle ...............................................................................679 

Gjoka, Monika .....................................................................................574 

Glass, Katherine A. ............................................................................241 

Glenn, Jeffrey S. ..................................................................................191 

Glikin, Gerardo C. ..............................................................................632 

Glimm, Hanno ...........................................................................204, 274 

Glorioso, Joseph C. ............................................. 285, 478, 566, 591 

Gochuico, Bernadette ......................................................................437 

Goddard, Melissa .......................................................................... 33, 90 

Goddard, Melissa A. ..........................................................................617 

Goerdt, Sergij ......................................................................................204 

Goetzmann, Jason................................................................................... 4 

Gogel, Brian M. .........................................................................199, 624 

Goins, William F. ................................................. 285, 478, 591, 690 

Gold, Michael .......................................................................................285 

Goldman, Frederick ..........................................................................587 

Goldman, Yale .....................................................................................523 

Goldowitz, Daniel ..............................................................................115 

Goldsmith, Corinne...........................................................................743 

Golebiowski, Diane ...........................................................................387 

Gombash, S. E. .....................................................................................381 

Gombash, Sara E. .....................................................................386, 596 

Gomero, Elida .....................................................................................702 

Gonçalves, Cristine ...........................................................................342 

Gonçalves, Manuel ............................................................................347 

Gong, Wenhui ......................................................................................369 

Gong, Zi ..................................................................................................495 

Gonzalez, Rodolfo....................................................................137, 465 

Gonzalez-Garay, Manuel .................................................................107 

Gonzalez-Gonzalez, Emilio ............................................................255 

Goodyer, Paul ......................................................................................247 

Gopal, Vijaya ..............................................................................150, 156 

Gordon, Myra E. .................................................................................575 

Gorham, Taron C. .................................................................................... 9 

Gorman, Kristen ................................................................................524 

Gorman, Wesley .................................................................................260 

Gorman, Wesley C. ............................................................................262 

Gornalusse, German ............................................................................81 

Gorsky, Joshua ....................................................................................544 

Goss, James R. .....................................................................................478 

Gosset, David .......................................................................................342 

Goto, Naoko .........................................................................................642 

Gottschalk, Stephen................................................................396, 400 

Gottschalk, Stephen M. ...................................................................410 

Gouble, Agnes .....................................................................................401 

Gouble, Agnès .....................................................................................731 

Goudy, Kevin ........................................................................................441 

Goudy, Kevin S. ...................................................................................323 

Gounis, Matthew................................................................................387 

Govindarajan, Srinath .....................................................................150 

Govindasamy, Lakshmanan ................................................334, 335 

Grabinski, Tessa .................................................................................596 

Gralow, Julie .........................................................................................303 

Grand, Francis .....................................................................................508 

Grandi, Paola .............................................................................416, 566 

Grange, Robert ......................................................................................90 

Grange, Robert W. ................................................................................33 

Gray, John T. .......................................................... 308, 671, 672, 702 

Gray, Steven J. ......................................................................................112 

Gray-Edwards, H. L. .............................................................................40 

Gray-Edwards, Heather ........................................................590, 592 

Gray-Edwards, Heather L....................................................... 36, 377 

Greco, Dario .........................................................................................501 

Greenberger, Joel S. ..........................................................................239 

Greene, Amy ........................................................................................657 

Greene, Michael R. ............................................................................527 

Gregory, Philip D. ............................................................ 46, 107, 183, 

......................................................184, 486, 487, 488, 504, 568, 678 

Grez, Manuel ..........................................................................................66 

Grieger, Joshua C. ..................................................................................54 

Grifin, Danielle ..................................................................................171 

Grifin, Danielle A. ..................................................................... 91, 172 

Grifiths, Duncan ...............................................................................264 

Grilley, Bambi .............................................................................. 14, 299 

Grimm, Dirk ............................................... 122, 123, 514, 516, 725 

Grizzle, William E. .............................................................................560 

Grody, Wayne W. ................................................................................497 

Grompe, Markus ..........................................85, 316, 466, 579, 581 

Grose, William E. ...............................................................................172 

Grossardt, Christian .........................................................................405 

Grosse, Stephanie ..............................................................................401 

152 



Grosso, Chantal ..................................................................................469 

Gruber, Harry ......................................................................................703 

Gruber, Harry E. .................................................................................676 

Gruenert, Dieter C. ....................................................... 471, 472, 575 

Gruh, Ina ...............................................................................................320 

Grunseich, Christopher...................................................................378 

Gu, Angel ...............................................................................................668 

Gu, Weiwang ........................................................................................533 

Guan, Xuan ...........................................................................................473 

Guan, Yue ..............................................................................................200 

Gubrij, Igor B. ......................................................................................439 

Guckes, Susan .....................................................................................295 

Guedan, Sonia .....................................................................................623 

Guedon, Jean-Marc ...........................................................................591 

Güenechea, Guillermo .....................................................................678 

Guenzel, Adam J. ................................................................................583 

Guenzel, Adam ....................................................................................748 

Guevara, Bruno ..................................................................................534 

GuhaSarkar, Dwijit ............................................................................212 

Guilak, Farshid ...................................................................................241 

Guilbaud, Mickaël ..............................................................................111 

Guilbault, Claire .................................................................................135 

Gül-Uludag, Hilal ...............................................................................462 

Gunther, Roland .................................................................................102 

Guo, Junjing..........................................................................................344 

Guo, Peixuan ........................................................................................515 

Gupta, Minnal ............................................................................298, 508 

Guschin, Dmitry Y. .............................................................................107 

Guschin, Dmitry .................................................................................184 

Guse, Kilian ................................................................................501, 607 

Guse, Kilian ..........................................................................................730 

Gutmann, Theresia ...........................................................................730 

Gutova, Margarita .............................................................................648 

Guy, John ...............................................................................................385 

Guzman-M, Karina ............................................................................564 

Haber, Tom ...........................................................................................719 

Habib, N.A. ............................................................................................682 

Hacein-Bey-Abina, Salima .......................62, 279, 306, 310, 313 

Hacke, Katrin .......................................................................................530 

Hackett, Neil R. ................................................................. 11, 106, 373 

Hackett, Perry .....................................................................................650 

Hackett, Perry B. .............................................................. 10, 102, 447 

Hadac, Elizabeth M. ..........................................................................407 

Haddad, Daniel J. ...............................................................................369 

Haddad, Marie Reine .............................................................383, 586 

Haemmerle, Reinhard .....................................................................422 

Haeusl, Martin ....................................................................................557 

Hafez, I. ..................................................................................................708 

Haft, Annelise......................................................................................316 

Haga, Kazunori ......................................................................................70 

Hagemann, Thorsten .......................................................................507 

Haggarty, Beth .......................................................................................46 

Hajri, Amor ..........................................................................................655 

Hakim, Chady H. ................................................. 166, 175, 295, 613 

Halama, Niels ......................................................................................405 

Halbert, Christine L. ...............................................................117, 443 

Hall, Bryan C. .......................................................................................447 

Halonen, Paavo J. ..................................................................................97 

Haluska, Jr, Paul .....................................................................................17 

Hamada, Hirofumi ............................................................................643 

Hamada, Katsuyuki ..........................................................................627 

Hamazaki, Takashi ............................................................................545 

Hamid, O. ..................................................................................................15 

Hamilton, Sara E. ...............................................................................270 

Hammer, Caroline ........................................................................ 33, 90 

Hammerschmidt-Kamper, Christiane ......................................725 

Hampson, David R. ...........................................................................390 

Hamzaoui-Nord, Silke .....................................................................405 

Han, Hye-Eun ......................................................................................380 

Han, Jingfen..........................................................................................248 

Han, Joohee ................................................................................408, 414 

Han, Joohee ................................................................................705, 742 

Han, Jun-Ming .....................................................................................720 

Han, Moon-Hee ..................................................................................143 

Han, Na ..................................................................................................151 

Han, Sang W. ................................................................... 265, 582, 658 

Han, Sung Yeol ....................................................................................375 

Han, Su-Youne .....................................................................................722 

Han, Zongchao ....................................................................................344 

Handy, Chalonda R. ...........................................................................287 

Hannigan, Timothy ...........................................................................611 

Hansen, C. .............................................................................................708 


153


Hansen, Gesine ......................................................................................84 

Hanson, Leah R. .................................................................................364 

Haodi, A. ................................................................................................682 

Happle, Christine ..................................................................................84 

Harbottle, Richard P. ..............................................................346, 354 

Hardcastle, Jayson J. ................................................................. 23, 641 

Harding, Cary O. .................................................................................354 

Harding-Smith, Rebekka E. ...........................................................460 

Hareendran, Sangeetha ..................................................................331 

Harel, Asaff ...........................................................................................478 

Hargrove, Phillip W................................................................... 26, 424 

Harmon, John W. ................................................................................616 

Harper, Jane ................................................................................. 59, 508 

Harper, Scott ........................................................................................295 

Harper, Scott Q..........................................................................171, 182 

Harrington, Kevin ........................................................... 18, 395, 652 

Hart, Stephen L. ............................................................ 158, 434, 435 

Hartemink, Alexander J. .................................................................241 

Hartig, Jörg S. ......................................................................................554 

Hartmann, Lynn ....................................................................................17 

Hartweck, Linn ...................................................................................169 

Harvey, Brandon K. ...........................................................................382 

Hasegawa, Mamoru ..................................................... 237, 432, 442 

Hasegawa, Naoyuki ..........................................................................643 

Hashimoto, Tadafumi ......................................................................283 

Hashimoto-Tamaoki, Tomoko ............................................218, 638 

Haskins, Mark .....................................................................................360 

Haskins, Mark E. ...................................................................................87 

Haso, Waleed .......................................................................................325 

Hassan, Namir .......................................................................................59 

Hassan, Namir J. .................................................................................508 

Hata, Taishi ..........................................................................................749 

Hatley, Mark E. ....................................................................................566 

Hauck, Bernd.............................................................................529, 547 

Haugwitz, Michael ............................................................................532 

Haurigot, Virginia .............................................................................488 

Hauser, Michael ..................................................................................295 

Hauswirth, William W. ..............................55, 115, 223, 545, 596 

Havlicek, David F. .................................................................................11 

Hayashita-Kinoh, Hiromi .....................................................120, 608 

Haynes, Russell D. .............................................................................255 

He, Bo .....................................................................................................367 

He, Chenxia ..........................................................................................731 

He, Ran ....................................................................................4, 326, 512 

He, Xianghui ..............................................................................207, 404 

He, Zhijian ............................................................................................691 

Hearl, William G. ................................................................................186 

Hebeler, Robert ..................................................................................624 

Heczey, Andras ......................................................................................12 

Heffernan, Thomas P. .......................................................................503 

Heffner, Garrett C. ...................................................................251, 258 

Heffner, Garrett .....................................................................................37 

Heiland, Teri L. ...................................................................................186 

Heinz, Niels ................................................................................320, 429 

Heiss, Christian ..................................................................................369 

Heiss, Kirsten ......................................................................................725 

Heitfeld, Suzanne ..............................................................................270 

Heldermon, Coy D. ............................................................................332 

Heller, Glenn ...........................................................................................61 

Heller, Kristin N. ............................................................... 91, 172, 291 

Heller, Richard .......................................................................................99 

Hemming, Richard ............................................................................379 

Hemminki, Akseli ............................................... 501, 680, 704, 730 

Hendry, Jason ......................................................................................593 

Henley, Jill E. ........................................................................................183 

Henley, Jill ...................................................................................184, 487 

Henley, Tom .........................................................................................551 

Henson, Karissa .................................................................................500 

Hentzschel, Franziska .....................................................................725 

Heo, Jeong ........................................................................................ 19, 22 

Hergenrother, Paul J. ........................................................................403 

Hermann, Alex ....................................................................................655 

Hermetet, Gregory ...............................................................................50 

Hernandez, Frank J. ..........................................................................509 

Hernandez, Luiza I. ...........................................................................509 

Herschman, Harvey R. .....................................................................448 

Hershield, Michael ..........................................................................307 

Herzog, Roland W. .............................................. 109, 190, 446, 669 

Heslop, Helen E. ............................................................ 299, 305, 398 

Hess, Richard ......................................................................................437 

Hetzel, Miriam ....................................................................................429 

Hew, Paggy ...........................................................................................403 

154 



Hickey, Ray D. ......................................................................................579 

Hickman, Suzanne E. .......................................................................304 

Hickman, Theresa ................................................................................19 

Hicks, Martin J. ........................................................................... 11, 727 

High, Katherine A. ..................... 8, 110, 488, 498, 529, 547, 733 

Higuchi, Takashi.......................................................................365, 753 

Hilje, Roman ........................................................................................320 

Hillestad, Matthew L. .............................................................583, 748 

Hinderer, Christian J.........................................................................110 

Hirai, Yukihiko ....................................................................................601 

Hiramoto, Takafumi ............................................................................86 

Hiraoka, Kei ................................................................................. 70, 522 

Hirata, Roli ..............................................................................................81 

Hiriyanna, Suja ...................................................................................709 

Hironaka, Kohei .................................................................................601 

Hiroshima, Kenzo ..............................................................................216 

Hirsch, Matthew L. ............................................................................268 

Hirschberg, Henry ............................................................................220 

Hirst, Rob A. .........................................................................................435 

Hirvinen, Mari ....................................................................................501 

Hishikawa, Shuji ................................................................................121 

Hitt, Mary ..............................................................................................403 

Ho, Ivy A. W. .........................................................................................254 

Ho, Michelle .........................................................................................492 

Hoeben, Rob C. ...................................................................................313 

Hogrel, Jean-Yves ...............................................................................734 

Holczbauer, Agnes.............................................................................696 

Holditch, Sara J ...................................................................................746 

Hoffman, Brad E. ................................................................................190 

Hoffman, Lauren ................................................................................648 

Hoherr, Sean E. ........................................................................481, 583 

Hogarth, Michael ...............................................................................161 

Hogg, Claire ..........................................................................................435 

Holczbauer, Agnes.............................................................................531 

Holder, Melanie N. ................................................................................33 

Hole, Patrick ........................................................................................264 

Holley, Lenton .....................................................................................660 

Hollis, Roger P. ............................................................................ 26, 492 

Holmes, Michael C. .....................................46, 107, 183, 184, 486, 

.......................................................................... 487, 488, 504, 568, 678 

Holt, Robert A. ....................................................................................115 

Holy, Jon ................................................................................................657 

Holzbaur, Erika L. F. ..........................................................................116 

Hom, Kellie ..............................................................................................44 

Hoon, Pak Seoung .............................................................................699 

Hope, Bruce T. .....................................................................................382 

Horkay, Iren .........................................................................................147 

Horvath, Staci .....................................................................................479 

Hosseini, Sayed Mohammd ...........................................................616 

Hotta, Akitsu .............................................................................357, 715 

Houghton, Frank ................................................................................239 

Howard, Carolann .............................................................................110 

Howard, Douglas B. ..........................................................................382 

Howe, Annett .........................................................................................48 

Howe, Steven J. .............................................................. 434, 435, 678 

Howell, Craig A. ..................................................................................509 

Howes, Rob ..........................................................................................245 

Hoxie, James A. ......................................................................................46 

Hoyos, Valentina ......................................................................392, 394 

Hoyos, Valentina ................................................................................684 

Hsieh, Matthew .......................................................................... 30, 428 

Hsieh, Pei-fang ....................................................................................723 

Hsu, David ..................................................................................474, 476 

Hu, Chuhong ........................................................................................497 

Hu, Huimin ...........................................................................................702 

Hu, Jim....................................................................................................433 

Hu, Ruozhen ........................................................................................712 

Hu, Yun...................................................................................................550 

Hu, Zebin ...............................................................................................555 

Hua, Giau ...............................................................................................474 

Hua, Kevin L............................................................................................46 

Huang, George ....................................................................................285 

Huang, Jeremiah ................................................................................287 

Huang, K.W. ..........................................................................................682 

Huang, Liang .......................................................................................457 

Huang, Patricia .........................................................................474, 476 

Huang, Wenlin ....................................................................................194 

Huang, Yuping.....................................................................................574 

Huard, Johnny ........................178, 179, 250, 281, 290, 611, 614 

Hubbard, S. M. ........................................................................................40 

Huch, Meritxell ......................................................................................85 

Hudecek, Michael ..............................................................................744 


155


Hudry, Eloise .............................................................................283, 540 

Hudson, J. A. ............................................................................................40 

Huffaker, Thomas B. .........................................................................712 

Hughes, Jonathan ....................................................................507, 561 

Hui, Daniel H. ......................................................................................110 

Huls, Helen ...........................................10, 198, 431, 502, 649, 650 

Hulsker, Margriet ..............................................................................609 

Humbert, L. ..........................................................................................694 

Hung, Chien-Ya ...................................................................................723 

Hung, Ming-Chuan ............................................................................723 

Hung, Tsuing-Jeu ...............................................................................723 

Hunter, David W. ................................................................................102 

Hurton, Lenka V. ......................................................................502, 621 

Hurwitz, Brian ....................................................................................747 

Hurwitz, Mary Y. ................................................................................131 

Hurwitz, Richard L. ..........................................................................131 

Hussain, Syed-Rehan A. ..................................................................171 

Hustedt, Jason ........................................................................................44 

Huston, Marshall W. ...............................................................275, 521 

Hutnick, Natalie A. ...............................................................................45 

Hutnick, Natalie .................................................................................341 

Hwang, M. ................................................................................................40 

Hwang, Misako ........................................................................... 36, 592 

Hwang, Tae-Ho .............................................................................. 19, 22 

Hwang, Yihharn .................................................................................106 

Hwu, Patrick ........................................................................................270 

Hwu, Wuh-Liang ........................................................................ 60, 376 

Hyang, Kim Ji .......................................................................................699 

Hyde, Stephen C. ................................................................................460 

Hyland, Kendra A. .............................................................................102 

Hyman, Bradley T. .............................................................................283 

Hyman, Bradley .................................................................................540 

Hyodo, Ichinosuke ............................................................................643 

Iankov, Ianko D. ..................................................................................641 

Iankov, Ianko .......................................................................................417 

Ibanez, Carlos .....................................................................................676 

Ibañez, Carlos .....................................................................................703 

Ida, Hiroyuki ........................................................................................365 

Ida, Hiroyuki ........................................................................................753 

Idirene, Idir ..........................................................................................399 

Ido, Hiroyuki .......................................................................................651 

Igarashi, Tsutomu .............................................................................228 

Iglesias, Diana .....................................................................................247 

Ihara, Hirotaka ...................................................................................150 

Iijima, Osamu ......................................................................................228 

Iinuma, Shin............................................................................................80 

Iizuka, Sayoko .....................................................................................365 

Ikawa, Eriko ............................................................................................80 

Ikawa, Yasuhiro ..................................................................................437 

Ikeda, Hiroaki ........................................................................................16 

Ikeda, Yasuhiro ......................................................................................68 

Ikeda, Yasuhiro .........................................................................726, 746 

Ikemoto, Tomokazu ..........................................................................121 

Ilett, Elizabeth .......................................................................................18 

Ilia, Fishbein ........................................................................................144 

Ilmarinen, T .........................................................................................732 

Imbrogno, Kara ..................................................................................290 

Imbrogno, Kayla ................................................................................611 

Impey, Helen ...........................................................................................48 

Inagaki, Aki ..........................................................................................703 

Inagaki, Akihito .......................................................................... 70, 522 

Ingham, Sheila J. M............................................................................265 

Inoue, Hiroyuki ......................................................69, 432, 440, 442 

Inoue, Makoto ................................................................ 237, 432, 442 

Inoue, Tomoko ....................................................................................442 

Iqball, Sharifah ......................................................................................56 

Irons, Eric E. ........................................................................................477 

Ishibashi, Hidetoshi .........................................................................120 

Ishii, Masaru ...........................................................................................80 

Islam, Saleem .........................................................................................57 

Ito, Hiroshi ...........................................................................................627 

Ito, Tomoko ..........................................................................................627 

Ivan, Alferiev S. ...................................................................................144 

Ivanovska, Irena I. .............................................................................523 

Ivics, Zoltan ..........................................................................................347 

Iwamoto, Takeo ..................................................................................585 

Iyombe, Jean-Paul ...................................................................293, 389 

Izsvak, Zsuzsanna .............................................................................347 

Izuka, Sayoko ......................................................................................585 

Jackson, Mark W.................................................................................276 

Jackson, Rineka .....................................................................................10 

Jacob, Aurélien ......................................................................................52 

156 



Jacobs, Edwin H. ................................................................................275 

Jagadeesh, Jayashree ........................................................................307 

Jäger, Dirk ...................................................................................405, 639 

Jahr, Holger ..........................................................................................275 

Jakobsen, Bent ....................................................................................508 

Jakobsen, Bent K. ....................................................................... 59, 298 

Jamet, Thibaud ....................................................................33, 90, 605 

Janas, Michelle L. ...............................................................................209 

Jang, Jae-Hyung ....................................................................................... 3 

Janssen, Paul M. L. .............................................................................291 

Janumala, Hadassah .........................................................................160 

Jares, Alexander ....................................................................................29 

Jarjour, Jordan ................................................................ 188, 193, 318 

Jaulin, Nicolas .....................................................................................111 

Javier, Francisco .................................................................................678 

Jay, Christopher M. ................................... 206, 272, 464, 510, 625 

Jayandharan, Giridhara R....................................................114, 328, 

................................................................331, 337, 536, 537, 542, 549 

Jena, Bipulendu .....................................................................................10 

Jensen, Michael C. ..............................................................................744 

Jensen, S ................................................................................................682 

Jeon, Daejong ......................................................................................602 

Jeong, Eu Gene ....................................................................................565 

Jeong, Hwa Yeon ................................................................................159 

Jeong, Ji Hoon ............................................................................349, 572 

Jerome, Keith R. ............................................................ 187, 188, 193 

Jessup, John M. ...................................................................................217 

Ji Hoon, Jeong ......................................................................................573 

Ji, Qingzhou ..........................................................................................243 

Jiang, Shuxia ........................................................................................511 

Jiang, Wei ..............................................................................................194 

Jiang, Yuanyuan ..................................................................................216 

Jiang, Yuhang .......................................................................................691 

Jiang, Zhong .........................................................................................512 

Jiang, Ziyue Karen .............................................................................215 

Jiao, L.R. .................................................................................................682 

Jie, Feilong ..................................................................................221, 563 

Jimenez-Orozco, Alejandro ...........................................................564 

Jin, Sheng-Yu ........................................................................................312 

Jing, Yuqi ...............................................................................................637 

Joglekar, Alok V. ........................................................................492, 571 

Johana, Nuryanti ......................................................................363, 580 

Johnson, Aaron ......................................................................................23 

Johnson, Aime K. ....................................................36, 377, 590, 592 

Johnson, Christopher .......................................................................660 

Johnson, J. A. ...........................................................................................40 

Johnson, Larry G. ...............................................................................439 

Johnson, Mai ........................................................................................215 

Johnston, Jennifer M. .......................................................................528 

Johnston, Sandra K. .............................................................................25 

Jolly, Douglas J...........................................................................676, 703 

Jone, Takako ...........................................................................................93 

Jones, Jeanne .......................................................................................351 

Jones, Pamela ......................................................................................139 

Jones, Steven J. M. ..............................................................................115 

Jones, Tara ............................................................................................473 

Jonlin, Erica .............................................................................................27 

Jordon, Andrea P. ..................................................................................46 

Joséphine, Charlène .........................................................................225 

Joseph, Glorioso C. ............................................................................416 

Joseph, Lauren ....................................................................................656 

Jossot, Laurent....................................................................................399 

Jost, Laurent ........................................................................................261 

Joubert, Romain ..............................................33, 90, 240, 605, 606 

Joung, J. Keith ............................................................................490, 738 

Jubeli, Emile .........................................................................................755 

Juckem, Laura .....................................................................................454 

Jund, Michelle D. ................................................................................270 

June, Carl H. .............................................................59, 298, 508, 623 

Jung, Hyesook .....................................................................................151 

Jung, Keun-Hwa ............................................................ 143, 598, 602 

Jung, Seungmoon ...............................................................................602 

Jung, Ulrike ..........................................................................................456 

Kabadi, Ami M. .........................................................................170, 491 

Kabanov, Alexander V. ...........................................................691, 693 

Kabasakal, Levent .............................................................................452 

Kaeppel, Christine.............................................................................274 

Kaestner, Klaus ...................................................................................581 

Kagele, Dominique A. ......................................................................712 

Kahn, Jason .............................................................................................44 

Kakarla, Sunitha.................................................................................400 

Kalaivani, V. ................................................................................536, 537 


157


Kaler, Stephen G. ......................................................................383, 586 

Kaliberov, Sergey A. ..........................................................................560 

Kaliberova, Lyudmila N. .................................................................560 

Kalinin, N. .............................................................................................724 

Kalle, Christof von .............................................................................210 

Kalos, Michael ................................................................... 59, 298, 508 

Kalra, Mamta .......................................................................................396 

Kamijima, Shuichi ..................................................................... 70, 522 

Kaminoh, Yuuki J. ..............................................................................180 

Kühn, Anna Luisa ..............................................................................387 

Kaminsky, Stephen M. ...............................11, 106, 373, 589, 727 

Kan, Shih-hsin .....................................................................................586 

Kanaan, Nicholas M. .........................................................................596 

Kaneda, Yasuhumi ...............................................................................80 

Kaneshiro, Kayleigh .........................................................................709 

Kang, Dae Hwang .................................................................................22 

Kang, Elizabeth ..................................................................................308 

Kang, Eunah .........................................................................................634 

Kang, Kevin ..........................................................................................574 

Kang, Robert ........................................................................................290 

Kang, Soyeong ....................................................................................375 

Kankaldyyan, Vazgen .......................................................................648 

Kannan, Vivekanandan ...................................................................508 

Kao, Chinghai .........................................................................................70 

Kapoor, Neena ....................................................................................307 

Kapp, Kerstin ......................................................................................686 

Kapranov, Philipp ..............................................................................681 

Karikó, Katalin ..........................................................................146, 147 

Karlen, Andrea D. ..............................................................................495 

Karli, Eerika .........................................................................................501 

Karponi, Garie........................................................................................61 

Karponi, Garyfalia ..................................................................... 27, 427 

Kasahara, Noriyuki ...... 70, 218, 522, 530, 638, 676, 682, 703 

Kasahara, Yuko N. ..............................................................................176 

Kashiwakura, Yuji ..............................................................................237 

Kaspar, Roger ......................................................................................652 

Kassner, Paul .............................................................................339, 550 

Katakowski, Joseph ..........................................................................205 

Kataoka, Kazunori ............................................................................350 

Kato, Kazunori ....................................................................................216 

Katz, Michael G. ..................................................................................374 

Kaufman, Michael L. ............................................................................26 

Kaufmann, Johanna K. .....................................................................554 

Kaufmann, Kerstin B. ..........................................................................66 

Kaur, Balveen ............................................................................409, 747 

Kauss, M. Ariel ....................................................................................542 

Kawabata, Masato ...................................................................192, 654 

Kawalekar, Omkar U. ........................................................................623 

Kawamura, Kiyoko ............................................................................216 

Kawano, Yasuhiro.................................................................................78 

Kawashima, Rei ..................................................................................643 

Kay, Mark A. ........................................... 7, 191, 271, 277, 343, 436 

Kaye, Frederic J. .................................................................................335 

Kean, Leslie ..........................................................................................650 

Kearns-Jonker, Mary ........................................................................468 

Kebriaei, Partow ...................................................................................10 

Keech, Naomi .........................................................................................48 

Keilman, George W. ..........................................................................140 

Kelleher, Michelle ..............................................................................232 

Kells, Adrian P. ............................................................... 288, 594, 599 

Kemp, C. J. .............................................................................................381 

Kemp, Christopher H. ......................................................................386 

Kennedy, Douglas R. .........................................................................311 

Kenny, Gavin D. ...................................................................................158 

Keramaris, Elizabeth .......................................................................296 

Kesari, Santosh ...................................................................................676 

Keshavjee, Shaf ...................................................................................433 

Ketzer, Patrick .....................................................................................554 

Khalique, Nada Abdul ......................................................................755 

Khan, Amir S. ..................................................................... 45, 341, 397 

Khan, Iram .......................................................................... 32, 318, 673 

Khargharia, Sanjib ...............................................................................41 

Khati, Makobetsa ...............................................................................189 

Khattak, Shahryar .............................................................................322 

Khayter, Cyd .........................................................................................490 

Khokher, Zeshan ......................................................................152, 620 

Khosravi-Far, R. .....................................................................................15 

Kicenuik, Kristin ................................................................................139 

Kichaev, Gleb .......................................................................................153 

Kiem, Hans-Peter ........................................25, 249, 311, 421, 673 

Kienle, Eike ................................................................................122, 123 

Kikuchi, Yasuhi .....................................................................................80 

158 



Kildebeck, Eric ..........................................................................324, 489 

Kim, Byung Sun ..................................................................................602 

Kim, Chang Won ...................................................................................19 

Kim, Dae Wook ...................................................................................138 

Kim, Dae-Sung ....................................................................................593 

Kim, Dong-Eun ...................................................................................159 

Kim, Hak Kyun ....................................................................................436 

Kim, Hyesung ......................................................................................256 

Kim, Hyun Ah ......................................................................................717 

Kim, Hyun .............................................................................................703 

Kim, Jaesung ..............................................................................640, 645 

Kim, Jane ..................................................................................................42 

Kim, Jeong-Min .............................................................. 143, 598, 602 

Kim, Ji Hyang .......................................................................................722 

Kim, Jin Tac ..........................................................................................722 

Kim, Jin-Hee .........................................................................................598 

Kim, Joon-Hyung ..................................................................................75 

Kim, Joseph J. ........................................................................................... 9 

Kim, Kenneth ............................................................................183, 487 

Kim, Keun-Sik .....................................................................................159 

Kim, Kwang S. .....................................................................................756 

Kim, Manho ................................................................................143, 602 

Kim, Marie ............................................................................................287 

Kim, Mi Kyung ........................................................................................22 

Kim, Mikyung ......................................................................................151 

Kim, Minjung .......................................................................................645 

Kim, Na Hyung ....................................................................................711 

Kim, Nak Won ...........................................................................349, 572 

Kim, Pyung-Hwan .............................................................................645 

Kim, Seung Geun...................................................................................22 

Kim, Seung U. ......................................................................................756 

Kim, So-Yoon ........................................................................................598 

Kim, Su Jeong ......................................................................................138 

Kim, Sung Wan .............................................................. 634, 640, 645 

Kim, Sung Wan .........................................................................685, 717 

Kim, Tae Won ......................................................................................565 

Kim, Tae-il ............................................................................................645 

Kim, Taekyun ......................................................................................151 

Kim, Yoon Jun ......................................................................................233 

Kim, Yoon S. .........................................................................................672 

Kimura, Takahiro ..................................................................................70 

Kincaid, Tiffany ..................................................................................529 

Kinchington, Paul R. .........................................................................591 

King, Christiaan D. ...............................................................................43 

King, Glenn ...........................................................................................139 

King, Oliver .............................................................................................93 

Kinnon, Christine ..............................................................................678 

Kinoh, Hiromi H. ................................................................................176 

Kipps, Thomas H. ..............................................................................502 

Kirn, David H. ................................................................................. 19, 22 

Kiroyan, Jason B. ................................................................................468 

Kishnani, Priya ...................................................................................276 

Kitagawa, Koichi ................................................................................192 

Kitaura, Kumiko .................................................................................150 

Kitchen, Scott G. ....................................................................................48 

Kizzire, Koby ..........................................................................................41 

Kleuss, Christiane .............................................................................686 

Klimczak, Ryan R. ................................................................................... 5 

Klingelhutz, Aloysius .......................................................................587 

Kloss, Christopher C. ........................................................................728 

Knight, Scott W. ..................................................................................243 

Knott, Christine ..................................................................................153 

Knovich, Mary Ann ...........................................................................253 

Knox, Van W. ...........................................................................................87 

Knutson, Keith ............................................................................ 17, 652 

Kobayashi, Hiroshi ..................................................................365, 585 

Kobayashi, Hiroshi ............................................................................753 

Kobayashi, Masao .................................................................................86 

Kobinger, Gary P. ................................................................................379 

Kocak, E. ................................................................................................695 

Koch, Walter ........................................................................................444 

Kochanek, Stefan .....................................................................126, 484 

Koeberl, Dwight D. ..................................................................276, 618 

Kofie, Robert M. ................................................................................283 

Kohn, Donald B.............................................26, 307, 492, 494, 571 

Kohnke, Monica .................................................................................224 

Kolarich, Andrew R. .........................................................................332 

Koldej, Rachel ........................................................................................48 

Komori, Junji ..........................................................................................79 

Konopka, Krystyna ...........................................................................576 

Koo, Taeyoung ....................................................................................734 

Koon, E. Colin ......................................................................................503 


159


Kopin, Etana ........................................................................................542 

Kornegay, Joe ..........................................................................................92 

Kortum, Amanda ...............................................................................419 

Kosasih, Paula .....................................................................................744 

Kostomitsopoulos, N. .......................................................................682 

Kottke, Timothy .....................................................18, 395, 505, 652 

Kotton, Darrell N. ..............................................................................107 

Kovacs, Krisztina ...............................................................................637 

Koya, Richard C. .................................................................................302 

Koyama, Yoshiyuki ............................................................................627 

Kozarsky, Karen .................................................................................364 

Kozlowski, Merek ..............................................................................418 

Kozlowski, Miroslaw ........................................................................236 

Kozyak, Benjamin..............................................................................391 

Kraeusslich, Hans-Georg ......................................................123, 514 

Kramer, Philipp ..................................................................................107 

Krammer, Peter H. .............................................................................204 

Kraus, Jan P. .........................................................................................583 

Krauss, Jürgen........................................................................................21 

Kreader, Carol A. ................................................................................243 

Kreppel, Florian ............................................................ 126, 134, 484 

Krishnagopal, Akshaya .........................................................331, 337 

Krishnamoorthy, Janani..................................................................431 

Krishnan, Aparna ..............................................................................476 

Krouse, Allen ............................................................................... 30, 428 

Kruck, Christa .....................................................................................379 

Krutzke, Lea ...............................................................................126, 484 

Kubo, Shuji ...................................................................... 216, 218, 638 

Kucharski, Cezary .............................................................................162 

Kuhn, Joseph .............................................. 199, 402, 503, 624, 625 

Kuhn, N. C .............................................................................................381 

Kuhn, Nathan C. .................................................................................386 

Kuhn, Nathan M. ................................................................................596 

Kuivanen, Antti ......................................................................................97 

Kulikovskaya, Irina .................................................................298, 508 

Kulkarni, Aditya .................................................................................745 

Kumar, Padmasini .........................199, 203, 206, 272, 317, 402, 

.................................................................................... 503, 624, 625, 633 

Kumar, Priti ..........................................................................................238 

Kumar, Sanjay .....................................................................................754 

Kumaresan, Pappanaicken ............................................................431 

Kumari, Monika..................................................................................549 

Kume, Akihiro ...........................................................................121, 651 

Kunkel, Hana ..........................................................................................66 

Kunkel, Louis M. ....................................................................................93 

Kuo, Chung-Jung ................................................................................253 

Kuo, Jennifer ........................................................................................215 

Kurosawa, Yoshikazu .......................................................................629 

Kurtzberg, Joanne ................................................................................62 

Kustikova, Olga S. ..............................................................................425 

Kutner, Robert ........................................................37, 251, 258, 674 

Kutner, Robert H. .....................................................................260, 262 

Kutschera, Ina .....................................................................................279 

Kuzmina, L............................................................................................724 

Kwak, Larry W. ...................................................................................626 

Kwiatkowski, Stanislaw ..................................................................714 

Kwon, Churl-Su ...................................................................................387 

Kwon, Minjung ...................................................................................151 

Kwon, Oh-Joon ....................................................................................634 

Kwon, Young Jik ............................................................... 42, 163, 220 

Kwong, King F. ....................................................................................513 

Kyba, Michael ............................................................................168, 169 

Kyeong, Lee Hyeon ...........................................................................699 

Kyuri, Lee ..............................................................................................573 

Lachmann, Nico ............................................................... 84, 320, 429 

Lad, Yatish ............................................................................................232 

Ladd, Bryan M. ....................................................................................102 

Laer, Dorothee von ...........................................................................195 

Lagasse, Eric ...........................................................................................79 

Lagresle-Peyrou, Chantal .....................................................313, 661 

Lai, Charles P. ......................................................................................103 

Lai, May C. ................................................................................................54 

Lai, Zhaohui .........................................................................................207 

Lake, John R. ........................................................................................102 

Lakshmipathy, Uma ..........................................................................244 

Lalezari, Jay .............................................................................................58 

Lam, Paula YP .....................................................................................254 

Lamb, Lawrence ................................................................................587 

Lamont, Jeffrey ...................................................................................624 

Landoli, Joseph .................................................................................676 

Landry, Breanne ................................................................................462 

Langlet-Bertin, Bérangère ................................................................52 

160 



Lankester, Arjen C. ............................................................................313 

Lapointe, M-O. ....................................................................................694 

Laprise, Stephanie ............................................................................115 

Lapteva, Natalia ....................................................................................14 

Larcher, Fernando .............................................................................347 

Larcher, Thibaut ................................................................................111 

Larochelle, Andre .................................................................................65 

Larosa, Fabrice ...................................................................................399 

Larsen, Helfe .......................................................................................755 

Latella, Maria Carmela ....................................................................347 

Lau, K.-H William ..............................................................................468 

Lauer, Anthony ...................................................................................454 

Laufs, Stephanie .......................................................................210, 518 

Laurent, Louise ..................................................................................465 

Lawlor, Michael W. .................................................................... 33, 605 

Lawlor, Michael .......................................................................... 90, 473 

Lawrence, Roger ................................................................................448 

Lawson, Lee Ann ..................................................................................57 

Lay, Frank .............................................................................................616 

Lazar, Daniel ........................................................................................189 

Lazarski, Christopher A. .................................................................737 

Le Clerre, Diane ..................................................................................401 

Le Guiner, Caroline .................................................................111, 734 

Le Roy, Florence .................................................................................294 

Le, Mickaël ...........................................................................................240 

Le, Steven Q..........................................................................................586 

Leaver, T. ................................................................................................708 

Lebel, Carl ................................................................................................83 

Leber, Mathias F. ................................................................................639 

Lebrun, J-J. ............................................................................................694 

Lebuhotel, Céline .....................................................................401, 683 

Lecomte, Christophe ...........................................................................52 

Lecomte, Emilie ....................................................................................76 

Ledent, Philippe .................................................................................261 

Lee, Albert ............................................................................................472 

Lee, Allyson ..........................................................................................283 

Lee, Amy ...................................................................................................44 

Lee, Brendan .............................................................................128, 607 

Lee, Bum Sup ......................................................................................565 

Lee, Dahye ............................................................................................238 

Lee, Daniel W. ......................................................................................325 

Lee, Dean A. .........................................................................................621 

Lee, Dean ..............................................................................................431 

Lee, Donghoon ....................................................................................443 

Lee, Doo Sung .....................................................................................640 

Lee, Edward .........................................................................................229 

Lee, Ellen K. .........................................................................................497 

Lee, Gary K. .............................................................................................58 

Lee, Gary ...............................................................................................183 

Lee, Gwang-Hyun ..............................................................................159 

Lee, Hong J. ..........................................................................................756 

Lee, Hyeon Kyeong ...........................................................................722 

Lee, Hyeon Youl ..................................................................................138 

Lee, Hyun Woo ...................................................................................722 

Lee, J. .......................................................................................................708 

Lee, Jae Chul ........................................................................................710 

Lee, Janet ..............................................................................................308 

Lee, Jangwook .....................................................................................238 

Lee, Jihyun ............................................................................................623 

Lee, Jong Young ..................................................................................143 

Lee, Jun Woo ...........................................................................................22 

Lee, Jung Eun.......................................................................................572 

Lee, Jung-Sun ......................................................................................130 

Lee, Ki Jeong ..............................................................................339, 550 

Lee, Kuen Yong ...................................................................................238 

Lee, Kwan Hee ....................................................................................565 

Lee, Kyung-Mi .....................................................................................654 

Lee, Kyuri ..............................................................................................349 

Lee, Lily .................................................................................................532 

Lee, Min Sang ............................................................................349, 572 

Lee, Minhyung ..........................................................................685, 717 

Lee, Narae .............................................................................................195 

Lee, Ni-Chung .............................................................................. 60, 376 

Lee, Sang Kun ............................................................................143, 602 

Lee, Sang-Kyung.................................................................................238 

Lee, Soonhee .......................................................................................151 

Lee, Soon-Tae ................................................................. 143, 598, 602 

Lee, Tao-Chen .....................................................................................723 

Lee, Won Jai .........................................................................................130 

Lee, Yeon Sook .......................................................................................19 

Lee, Youngsook...................................................................................685 

Lee, Yu Kyung .........................................................................................22 


161


Leen, Ann ..............................................................................................396 

Leen, Ann M. ........................................................................................398 

Leger, Adrien ..........................................................................................76 

Leger, Andrew J. .................................................................................453 

Léger, Thibaut .....................................................................................612 

Legg, Alison..........................................................................................508 

Lei-butters, Diana CM......................................................................535 

Lemaire, Laetitia ................................................................................401 

Lemoine, Nicholas ............................................................................561 

Lemoine, Nicholas R. .......................................................................507 

Lenarsky, Carl .....................................................................................203 

Leng, Qixin ..............................................................................................44 

Leo, Simone ...............................................................................309, 517 

Lerch, Thomas F. ................................................................................336 

Leroy, Stanley G. .................................................................................592 

Leslie, George J. .....................................................................................46 

Lesueur, Léa L. ....................................................................................148 

Letteri, Rachel .....................................................................................157 

Leung, A. ................................................................................................708 

Leung, Kitty .........................................................................................433 

Levin, Bernard R. ..................................................................................48 

Levine, Bruce L. ................................................................ 59, 298, 508 

Levy, Matthew.....................................................................................205 

Levy, Robert J. ................................................................ 144, 366, 372 

Levy, Robert .........................................................................................374 

Levy, Seth G. .........................................................................................548 

Li, Baozheng ..............................................................................541, 542 

Li, Brian ....................................................................................................29 

Li, Fanying ...............................................................................................99 

Li, Feng ...................................................................................................345 

Li, Haitang ............................................................................................668 

Li, Hojun ................................................................................................488 

Li, Hongmei ..........................................................................................715 

Li, Hongtao ...........................................................................................406 

Li, Hongwei ..................................................................... 221, 533, 563 

Li, Huapeng ............................................................................................... 4 

Li, Jia ........................................................................................4, 326, 512 

Li, Jianbin ....................................................................................367, 370 

Li, Jing.....................................................................................................155 

Li, Jinlong ..............................................................................................221 

Li, Juan .............................................................................. 367, 370, 449 

Li, Leping ..............................................................................................242 

Li, Li B. ...................................................................................................266 

Li, Liyuan ..............................................................................................136 

Li, Mengxin ............................................................................4, 128, 326 

Li, Pengpeng ........................................................................................255 

Li, Pingjuan ..........................................................................................467 

Li, Shaoyong .........................................................................4, 128, 326 

Li, Shulin ...............................................................................................139 

Li, Songtao ............................................................................................618 

Li, Tianbai .............................................................................................533 

Li, Tiansen ............................................................................................709 

Li, Wei .....................................................................................................752 

Li, Wenpeng .........................................................................................195 

Li, Xiaodan ............................................................................................404 

Li, Xiaomiao ...............................................................................328, 542 

Li, Xiaoming .........................................................................................664 

Li, Xuan S...............................................................................................107 

Li, Yan .....................................................................................................129 

Li, Yi ........................................................................................................640 

Li, Yonghai ............................................................................................529 

Li, Zongyi ..............................................................................................729 

Liadi, Ivan .............................................................................................393 

Liang, Zhibing .....................................................................................533 

Liao, Sophia H. Y. ................................................................................356 

Liao, Wei ................................................................................................107 

Lidonnici, Maria Rosa ......................................................................420 

Lieber, Andre .................................................13, 201, 303, 483,552, 

.......................................................................... 679, 680, 689, 704, 729 

Lifshitz, Samuel ..................................................................................503 

Liikanen, Ilkka ....................................................................................730 

Lim, In J. .................................................................................................756 

Lin, Amy ......................................................................................676, 703 

Lin, Chi-Chen .......................................................................................348 

Lin, Michael Z. .....................................................................................255 

Lin, P. .......................................................................................................708 

Lin, Yanni ....................................................................................489, 567 

Lin, Yun ........................................................................................339, 550 

Lin, Ziguang .........................................................................................474 

Lindqvist, Johan M. ...........................................................................173 

Lindsay, Cory R. ..................................................................................324 

Linette, Gerald P. ........................................................................ 59, 508 

162 



Ling, Changquan ....................................................75, 118, 314, 328 

Ling, Chen ........................ 75, 118, 314, 328, 333, 334, 541, 542 

Lipinski, Daniel M. ............................................................................229 

Lipshutz, Gerald S. ..................................................................497, 571 

Lipton, Jack W. ....................................................................................386 

Lisowski, Leszek .................................................................7, 191, 277 

Litterman, Adam J. ............................................................................270 

Little, Dianne .......................................................................................276 

Litzky, Leslie ...........................................................................................59 

Liu, C. ......................................................................................................682 

Liu, Chao Lien .....................................................................................670 

Liu, Daofeng ............................................................................................12 

Liu, Dexi .......................................................................................242, 750 

Liu, Enli ..................................................................................................299 

Liu, Guei-Sheung ......................................................................230, 740 

Liu, Guihua ...........................................................................................473 

Liu, Hao .................................................................................................300 

Liu, Jian ..................................................................................................295 

Liu, Lingfeng ........................................................................................744 

Liu, Lixin ...............................................................................................616 

Liu, Mingyao ........................................................................................433 

Liu, Pingguo .........................................................................................208 

Liu, Shi-he ...................................................................................272, 659 

Liu, Shu-Fen .........................................................................................723 

Liu, X.X. ..................................................................................................682 

Liu, Xiang ..............................................................................................219 

Liu, Xiu Y. ...............................................................................................509 

Liu, Xiuying ..........................................................................................511 

Liu, Yanxin ............................................................................................752 

Liu, Yijia .................................................................................................455 

Liu, Yuli .....................................................................................................87 

Liu, Yu-Ping .............................................................................................20 

Liu, Zhongda ........................................................................................214 

Livingston, Justin ...............................................................................208 

Llewellyn, Nick .........................................................................184, 487 

Lo, Mandy .............................................................................................322 

Loeb, Keith R. ......................................................................................140 

Logan, Grant J. .....................................................................................356 

Logg, Christopher R. ...................................................... 70, 522, 703 

Lombardo, Angelo ....................................................... 469, 486, 504 

Lomeli-R, Christian Y. ......................................................................564 

Lonard, David M. ...............................................................................459 

Long, Harry .............................................................................................17 

Lopez, Patricia ....................................................................................476 

Lopez, Veronica M. ............................................................................635 

Lorain, Stéphanie ..............................................................................225 

Lorincz, Matthew ..............................................................................322 

Lostal, William.......................................................................................98 

Lothrop Jr, Clinton D. .......................................................................498 

Louis, Chrystal ....................................................................................396 

Low, Walter C. ...........................................................................364, 495 

Lu, Aiping ..................................................... 178, 179, 250, 290, 611 

Lu, Jiamiao ............................................................................................271 

Lu, Maomin ..........................................................................................533 

Lu, Pei Juan ..........................................................................................296 

Lu, Peijuan ............................................................................................451 

Lu, Qi Long .................................................................................296, 451 

Lu, Rong....................................................................................................29 

Lu, Taihe ................................................................................................671 

Lu, Yi .......................................................................................................136 

Lu, Yuan ............................................................................... 75, 118, 335 

Lu, Yuanqing ........................................................................................545 

Lu, Zhi Hong ........................................................................................560 

Lu, Zhuo-zhuang ......................................................................644, 729 

Lucas, Cherry ......................................................................................232 

Lucke, Colleen .......................................................................................... 9 

Luke, Jeremy ........................................................................................185 

Lum, Lawrence ...................................................................................711 

Luna Velez, María Victoria.............................................................330 

Lundberg, Cathryn ...............................................................................99 

Luo, Jie .........................................................................................221, 563 

Luo, Jun .....................................................................................................96 

Luo, Xianghua .....................................................................................414 

Luo, Yuxia .............................................................................................338 

Lüttge, Doreen .......................................................................................84 

Lutzko, Carolyn ..................................................................................426 

Lyde, Randolph ....................................................................................... 9 

Lydie, Guigand ....................................................................................111 

Lyle, Meghan .......................................................................................670 

Lythgoe, Mark F. .......................................................................158, 434 

Ma, Hsin-I .............................................................................................720 

Ma, Wenqin ................................................................................541, 542 


163


Ma, Yongjie ...........................................................................................750 

Ma, Zhijun .............................................................................................671 

Mac, Helen ............................................................................................224 

MacAldaz, Margarita E. ...................................................................390 

MacDonald, Kelvin D. .......................................................................361 

Machen, Laurie .........................................................................141, 149 

Machluf, Marcelle ..............................................................................719 

Machuca, Tiago N. .............................................................................433 

Machuca-R, Catalina ........................................................................534 

Mack, David .........................................................................................473 

Mackall, Crystal L. .............................................................................325 

MacLaren, Robert E. .........................................................................229 

MacVicar, B. ..........................................................................................708 

Madinaveitia-V, Juan A. ...................................................................246 

Madoiwa, Seiji .....................................................................................121 

Madtes, David K. ................................................................................117 

Maeder, Morgan L. ............................................................................738 

Magee, Mitchell J. ...............................................................................624 

Maggio, Aurelio .....................................................................................61 

Magnani, Zulma..................................................................................504 

Magno, Aaron L. ....................................................................................54 

Maguire, A. ..............................................................................................53 

Maguire, Albert M. ..................................................................224, 227 

Maguire, Casey A. ......................................................... 103, 540, 751 

Maguire-Zeiss, Kathleen .................................................................457 

Mah, Cathryn S. .....................................................................................57 

Mahadevan, Lavanya ............................................................................. 9 

Mahajan, Vivek ...................................................................................693 

Mahdipoor, Parvin ............................................................................213 

Mahendravada, Aruna ............................................................. 49, 300 

Maiti, Sourindra N. ............................................................................626 

Maiti, Sourindra .......................................................................431, 502 

Maitland, Stacy ..........................................................1, 384, 387, 595 

Majka, Marcin .....................................................................................714 

Makkia, Rasha .....................................................................................344 

Malani, Nirav .............................................................................306, 310 

Male, Frances ......................................................................................310 

Malech, Harry L. ......................................................................... 82, 308 

Malerba, Alberto ................................................................................734 

Malik, Alock ................................................................................. 92, 391 

Malik, Punam ......................................................................................664 

Maltais, Chantale ..................................................................................83 

Malvilio, Fulvio ...................................................................................734 

Mamchaoui, Kamel ...........................................................................491 

Mandel, R. J. .........................................................................................381 

Mandel, Ronald J. .....................................................................386, 721 

Mandelli, Giacomo ............................................................................420 

Manfredsson, F. P. ..............................................................................381 

Manfredsson, Fredric P. ........................................................386, 596 

Mangena, Ramu .................................................................................551 

Maniati, Eleni ......................................................................................507 

Mann, Trenae ......................................................................................200 

Mannioui, Cécile Schiffer ...............................................................683 

Manoli, Irini .........................................................................................280 

Mao, Qin ................................................................................................512 

Mao, Sunnie .........................................................................................692 

Mao, Yingying .....................................................................................533 

Maples, Phillip B. ..................................... 199, 203, 206, 234, 272, 

....................... 352, 402, 464, 503, 510, 570, 577, 624, 625, 633 

Maranzano, Michael .........................................................................276 

Marchetti, Dario .................................................................................392 

Marcus, Nancy ....................................................................................211 

Mardini, Osama ..................................................................................103 

Marescau, Bart ...................................................................................497 

Margaritis, Paris ................................................................................733 

Margulies, Kenneth B. .....................................................................374 

Marinova, Ekaterina ............................................................................12 

Markert, Chad .....................................................................................473 

Marktel, Sarah ....................................................................................420 

Markusic, David .................................................................................669 

Markusic, David M. ...........................................................................109 

Marolleau, Beatrice .............................................................................76 

Marquez, Laura I. ...............................................................................466 

Marrocco, Elena .................................................................................539 

Mars, Wendy ........................................................................................611 

Marsh, A. ..................................................................................................15 

Marshall, K. .............................................................................................53 

Marsic, Damien ........................................................................335, 548 

Martell, L. A. ............................................................................................15 

Marti, Guy P. .........................................................................................616 

Martin, D. R. ............................................................................................40 

Martin, Daniel ........................................................................................57 

164 



Martin, Douglas R. .......................................36, 377, 387, 590, 592 

Martin, John .........................................................................................338 

Martin, Samia .......................................................................33, 90, 606 

Martinelli, Valentina ........................................................................368 

Martinez-F, Francisco ................................................. 246, 534, 564 

Maruyama, Kazuo ...................................................................142, 145 

Marzulli, Marco ........................................................................416, 566 

Massie, Bernard .................................................................................135 

Mastaglio, Sara ...................................................................................504 

Masurier, Carole ............................................................................ 33, 90 

Mata, Melinda .....................................................................................400 

Matalon, Reuben ...................................................................................35 

Matar, Majed ........................................................................................461 

Matern, Dietrich .................................................................................583 

Matrai, Janka .......................................................................................496 

Matrobattista, Enrico ......................................................................707 

Matsui, Hideto ....................................................................................357 

Matsumoto, Priscila K. ..........................................................582, 658 

Mattar, Citra .................................................................... 363, 430, 580 

Matthews, Carolyn M. ......................................................................503 

Mattoo, Abid ........................................................................................217 

Maus, Marcela V. ...................................................................................59 

Mavilio, Fulvio ............................................................................ 76, 347 

Maynard, Ryan ....................................................................................525 

Mays, Lauren E. .....................................................................................71 

Mazarakis, Nicholas D. .......................................................................67 

Mazzacurati, Lucia ..................................................................416, 566 

Mazzieri, Roberta ..............................................................................301 

McAnulty, Robin J. .............................................................................434 

McBride, Jodi .......................................................................................284 

McCaffrey, Anton P. ...........................................................................454 

McCart, Andrea ..................................................................................561 

McCarty, Douglas ..................................................................................39 

McCarty, Douglas M. ...............................................................125, 597 

McClure, Diane ...................................................................................461 

McCown, Thomas J. ..........................................................................112 

McCraw, Dustin ..................................................................................336 

McCurdy, V. J. ..........................................................................................40 

McCurdy, Victoria J. ..............................................36, 377, 590, 592 

McDoom, Isaam ....................................................................................55 

McElroy, Amber N. ............................................................................738 

McGinley, Lisa M. ...............................................................................475 

McGinn, Arlo N. ..................................................................................685 

McGrath, John A. ................................................................................578 

McGrath, John ........................................................................................80 

McInerny, Simone C. .........................................................................115 

McIvor, R. Scott .................................................... 102, 364, 447, 495 

Mckay, Tristan R. ...............................................................................435 

McKenna, Robert ..................................................................................71 

McLeod, Cameron W. .......................................................................158 

McNeish, Iain ......................................................................................561 

McNiece, Ian ...........................................................................................10 

McVicar, Daniel W..............................................................................308 

Mead, Andrew ............................................................................ 92, 391 

Meadows, Aaron ........................................................................ 39, 597 

Medrano, Ruan Felipe V. .................................................................630 

Medzikovic, Lejla ...............................................................................369 

Mei, Zhuyong .......................................................................................299 

Melcher, Alan ...........................................................18, 395, 505, 652 

Melo, Sandra ........................................................................................277 

Memmott, Trevor ..............................................................................208 

Mendeleeva, L. ....................................................................................724 

Mendell, Jerry R. ....................................................91, 172, 291, 295 

Mendoza, Janess ................................................................................341 

Mendoza, Janess M. ..........................................................................153 

Meng, Xianmei ....................................................................................468 

Merchan, Jaime ...................................................................................637 

Merigan, William H. ............................................................................... 5 

Merkel, Christian A. ..........................................................................630 

Merkel, Olivia ......................................................................................711 

Merkert, Sylvia ......................................................................................84 

Merlin, Simone .........................................................................315, 469 

Merten, Otto W. .....................................................................................52 

Merwin, Miles J. ..................................................................................344 

Messaddeq, Nadia ........................................................ 240, 278, 605 

Metelitsa, Leonid ..................................................................................12 

Metzge, Mark ..........................................................................................30 

Metzger, G. ............................................................................................695 

Metzger, Joseph M. ............................................................................538 

Metzger, Mark .....................................................................................428 

Meunier, M. ..........................................................................................694 

Meyer, Nancy .......................................................................................336 


165


Mi, Tiejuan ...................................................................... 198, 502, 621 

Miao, Carol H. ....................................................... 101, 140, 665, 670 

Miao, Lei ................................................................................................691 

Michel, Gérard .......................................................................................62 

Midoux, Patrick ..................................................................................342 

Miest, Tanner S. ..................................................................................415 

Mikals, Kyle .............................................................................................71 

Mikkelsen, Tom ..................................................................................676 

Miko, Edit ..............................................................................................147 

Milazi, Stephanie ...............................................................................451 

Milczarek, Olga ...................................................................................714 

Miller, A. D. ...........................................................................................117 

Miller, Andrew ....................................................................................387 

Miller, Auston ......................................................................................168 

Miller, Christopher P. .......................................................................423 

Miller, Christophre P.........................................................................419 

Miller, Dusty ........................................................................................443 

Miller, Erin ............................................................................................542 

Miller, Jeffrey C. ..................................................................................184 

Miller, Jr, Francis J. .............................................................................511 

Miller, Timothy J. ...............................................................................500 

Millington, Michelle L. ........................................................................48 

Millroy, Laura ......................................................................................189 

Mills, Jason A. ......................................................................................224 

Mimuro, Jun .........................................................................................121 

Min Sang, Lee ......................................................................................573 

Mineno, Junichi ................................................................ 16, 629, 651 

Mingozzi, Federico ..................................................................110, 498 

Mingozzi, Frederico ............................................................................... 8 

Mintz, P.J. ...............................................................................................682 

Mintzer, Esther ...................................................................................188 

Mir, Lluis M. .........................................................................................148 

Miranda, Maria V. ..............................................................................632 

Mishra, Suparna .................................................................................307 

Miskin, James ......................................................................................232 

Mitchell, Erin .................................................................................. 33, 90 

Mitchell, James B. ..............................................................................743 

Mitchell, Marilyn...................................................................................92 

Mithbaokar, Pratibha .......................................................................553 

Mitrophanous, Kyriacos ......................................................... 56, 232 

Mitsuyasu, Ronald ................................................................................58 

Mittal, Suresh K. .................................................................................628 

Miura, Yoshi .........................................................................................414 

Miura, Yoshiaki .............................................................. 408, 705, 742 

Mixson, Archibald J. .............................................................................44 

Miyake, Koichi ................................................................ 228, 358, 601 

Miyake, Noriko .........................................................................228, 358 

Miyamoto, Shohei..................................................69, 432, 440, 442 

Mizandara, M. .....................................................................................682 

Mizoguchi, Yoko ....................................................................................86 

Mizuguchi, Hiroyuki .........................................................................482 

Mizukami, Hiroaki ..................................................................121, 651 

Mizushima, Tsunekazu ...................................................................749 

Moal, Christelle ...............................................33, 90, 240, 605, 606 

Moats, Rex A. .......................................................................................648 

Mochizuki, Shinji ..................................................................................86 

Modlich, Ute...............................................................................422, 425 

Moi, Davide ................................................................................301, 486 

Moisyadi, Stefan .................................................................................569 

Mok, Steven..........................................................................................302 

Mokhtari, Saloomeh .........................................................................253 

Monassier, Laurent ...........................................................................278 

Monette, Sébastien .................................................................373, 589 

Monjaret, François ............................................................................615 

Montazeri Aliabadi, Hamidreza ..................................................213 

Montgomery, Chrystal .....................................................................171 

Montgomery, Chrystal L. ........................................................ 91, 291 

Montiel-Martínez, Ana G. ...............................................................604 

Montini, Eugenio ..........................28, 63, 64, 233, 309, 517, 666 

Montus, Marie .....................................................................................734 

Moon, Jangsup ....................................................................................598 

Mooney, Michael ................................................................................378 

Moore, Malcolm A.S. .........................................................................727 

Moore, Theodore ...............................................................................307 

Mookherjee, Suddhasil ...................................................................709 

Moore, Andrea R. ...............................................................................681 

Mooreield, Emily ..............................................................................473 

Moreo, Andrew ..................................................................................182 

Morgan, Mary .........................................................................................65 

Morgan, Richard A. ...........................................................................716 

Mori, Masaki ........................................................................................749 

Moon, Anne ..................................................................................... 19, 22 

166 



Morishita, Naoya .....................................................................192, 654 

Moritz, Thomas ......................................................84, 320, 422, 429 

Morrison, Kyle P. ................................................................................140 

Morrison, Nancy ...................................................................................36 

Morrow, Matthew P .............................................................................45 

Moser, Anne ............................................................................................37 

Mosquea, Leocadia M. .....................................................................453 

Moss, Bernard .....................................................................................681 

Mota, Daniel ........................................................................................656 

Moughon, Diana .................................................................................215 

Moullier, Philippe .........................33, 76, 90, 111, 445, 606, 734 

Moulton, Hong M. ..............................................................................451 

Mrugala, Maciej M. ...............................................................................25 

Mu, Anbin .............................................................................................374 

Mu, Dakai ..............................................................................................540 

Mu, Dakai ..............................................................................................751 

Mu, Xiaodong ......................................................................................179 

Mucci, Adele ...........................................................................................84 

Mueller, Ann-Kristin ........................................................................725 

Mueller, Christian ..................................... 211, 361, 388, 603, 735 

Mueller, Martin ...................................................................................123 

Muenzer, Joseph .................................................................................495 

Muether, Nadine ................................................................................557 

Mukhopadhyay, Debabrata ...........................................................652 

Mulhern, Orla ......................................................................................184 

Mulhern, Orla L. .................................................................................487 

Müller-Kuller, Uta .................................................................................66 

Muñoz-Fuentes, Rosa M. ................................................................604 

Munye, Mustafa M. ..................................................................434, 435 

Muramatsu, Hiromi ................................................................146, 147 

Muramatsu, Shin-ichi .............................................................. 60, 376 

Murlidharan, Giridhar .......................................................................... 2 

Murrey, Darren ........................................................................... 39, 597 

Murugan, Dorai ..................................................................................754 

Mussolino, Claudio ...........................................................................539 

Muthumani, Karuppiah ............................................................. 9, 341 

Muthuswamy, Jit ................................................................................100 

Myers, Doug ............................................................................................14 

Myles, Devin J. ............................................................................. 45, 397 

Naash, Muna I. ....................................................................................344 

Naissi, Naiseh .........................................................................688, 713 

Nagabhushan Kalburgi, Sahana ..................................................112 

Nagamoto, Yasuhito .........................................................................482 

Nagasaki, Masao ...................................................................................86 

Nair, Nisha ....................................................................................... 8, 496 

Nair, Sukesh C. ....................................................................................114 

Najbauer, Joseph ................................................................................656 

Najjar, Amer .........................................................................................650 

Nak Won, Kim .....................................................................................573 

Nakade, Koji .........................................................................................643 

Nakai, Hiroyuki ...............................................72, 78, 119, 124, 259 

Nakajima, Yoshiyuki ............................................................................79 

Nakamura, Kazuhiro ...........................................................................86 

Nakamura, Masataka .......................................................................651 

Nakamura, Shin-ichiro ....................................................................482 

Nakanishi, Yoichi ...................................................69, 432, 440, 442 

Nakano, Yuki .......................................................................................440 

Nakano, Yuri ........................................................................................643 

Nakashima, Hiroshi ..........................................................................690 

Nakauchi, Hiromitsu ...................................................... 86, 365, 753 

Naldini, Luigi ........................................ 63, 64, 197, 233, 301, 309, 

......................................................................... 323, 441, 469, 486, 496, 

.................................................................................... 504, 517, 666, 667 

Nam, Hye Yeong .................................................................................645 

Nam, Hyun-Joo ......................................................................................71 

Nam, Jeong A. ......................................................................................572 

Nam, Kihoon ..............................................................................685, 717 

Nan, Zenhong ......................................................................................495 

Nan, Zhenhong ...................................................................................364 

Narayan, Poornima K. L. .................................................................267 

Nargarkhan, Simon ...........................................................................169 

Nariai, Naoki ..........................................................................................86 

Narukawa, Munetoshi .....................................................................530 

Narusawa, Megumi .................................................................432, 442 

Nathan, Erica .......................................................................................156 

Nathwani, Amit ........................................................................363, 580 

Naughton, Bartholomew J. .................................................... 39, 597 

Nawa, Akihiro .....................................................................................627 

Nayak, Ramesh ...................................................................................426 

Nayak, Sushrusha ..............................................................................446 

Naylor, Stuart .............................................................................. 56, 232 

Neal, Tawny L. .....................................................................................174 

Neduva, Victor ....................................................................................309 


167


Negishi, Yoichi ..........................................................................142, 145 

Negre, Olivier ......................................................................................674 

Neiswender, James............................................................................212 

Nektaria, Andronikou ......................................................................706 

Nelson, Carol A. ..................................................................................453 

Nelson, Jared .......................................................................................263 

Nemnaitis, John ..................................................................................317 

Nemunaitis, Derek ............................................................................273 

Nemunaitis, John J. ....................................15, 199, 203, 206, 219, 

...........................................234, 272, 273, 351, 352, 402, 459, 464, 

............................................479, 503, 510, 570, 577, 624, 625, 659 

Nerem, Robert M. ..............................................................................475 

Nettelbeck, Dirk .................................................................................483 

Nettelbeck, Dirk M. ................................................................... 21, 554 

Ng, Philip...............................................................................................353 

Ngo, Minhtran C. ................................................................................396 

Nguyen, Tran T....................................................................................690 

Nichol, Geoff ...........................................................................................58 

Nichols, Timothy ...............................................................................667 

Nicolay, Jan P. .......................................................................................204 

Nicolis di Robilant, Benedetta .....................................................197 

Nicholls, J.P. ..........................................................................................682 

Nichols, Timothy C............................................................................733 

Nicholson, David ................................................................................755 

Nicolson, Sarah C. .................................................................................74 

Nicolson, Tamara ..................................................................................48 

Nie, Daibang ........................................................................................281 

Niemeyer, Glenn P. ............................................................................498 

Nienhuis, Arthur W. ..........................................................................672 

Nienhuis, Arthur ................................................................................702 

Nieses, Amanda ..................................................................................468 

Nilsson, Bradley L. ...............................................................................43 

Nilsson, Jonas ......................................................................................321 

Nimgaonkar, Vishwajit ....................................................................239 

Niopek, Dominik ................................................................................514 

Nishikawa, Shinichi ..........................................................................753 

Nishimura, Junichi ............................................................................749 

Nitahara-Kasahara, Yuko .....................................................120, 608 

Nixon, Alan J. ...........................................................89, 257, 297, 718 

No, Daniel ...................................................................................152, 620 

Noble, Misty L. ..........................................................................101, 140 

Noh, Moon Jong ..................................................................................565 

Norris, James .......................................................................................219 

Norvell, Fabienne ....................................................................206, 633 

Noske, David P. ...................................................................................321 

Noskov, Alexander ............................................................................465 

Novicoff, Sarah .........................................................................497, 571 

Nowrouzi, Ali ............................................................................204, 274 

Noyes, Marcus B.................................................................................485 

Nunan, Robert ....................................................................................273 

Nurro, Jussi .............................................................................................97 

Nusco, Edoardo ..................................................................................353 

Nygaard, Sean C. ................................................................................316 

Oakes, Benjamin L. ...........................................................................485 

Obata, Yuichi .......................................................................................643 

Obeng-Adjei, Nyamekye ....................................................................45 

O’Callaghan, Christopher ...............................................................435 

Ochala, Julien ......................................................................................173 

O’Connell, Ryan M. ............................................................................712 

O’Connor, Colleen ..............................................................................650 

O’Connor, Colleen M. ........................................................................653 

O’Connor, Geraldine .........................................................................308 

Oda, Yusuke .........................................................................................145 

Odom, Guy L. .......................................................................................543 

O’Donnell, Patricia ............................................................................360 

O’Donnell, Patricia A. ............................................................... 87, 289 

Ogino, Atsushi .....................................................................................216 

Ogris, Manfred ....................................................................................134 

Oh, Eonju...............................................................................................129 

Oh, Il Ung ..............................................................................................375 

Oh, Jonathan ........................................................................................503 

Ohashi, Kazuo ............................................................................. 79, 237 

Ohashi, Toya ..............................................................................365, 585 

Ohashi, Toya ........................................................................................753 

Ohlfest, John R. ...................................................................................270 

Ohlin, K. Elisabet ...............................................................................631 

Ohlstein, Jason ....................................................................................444 

Ohmine, Ken ........................................................................................651 

Ohmori, Tsukasa ......................................................................121, 237 

Ohnami, Shumpei ..............................................................................642 

Ojeda, Sergio R. .....................................................................................78 

Okada, Hironori ............................................................ 120, 176, 608 

Okada, Takashi .............................................................. 120, 176, 608 

168 



Okamoto, Sachiko ..................................................................... 16, 629 

Okano, Teruo ............................................................................... 79, 237 

Old, Matthew .......................................................................................747 

Olivares, Simon ........................10, 198, 502, 621, 649, 650, 653 

Oliviera, Anna V. .................................................................................160 

Olsen, Curtis ........................................................................................685 

Olsen, John C. ......................................................................................535 

Olson, Erik R. .......................................................................................102 

Olson, Susan ........................................................................................466 

Olzzewska, Malgorzata ......................................................................50 

O’Malley, Bert W. ................................................................................459 

O’Malley, Thomas ..............................................................................360 

Omata, Daiki ........................................................................................145 

O’Neill, Kim ..........................................................................................208 

Orchard, Paul .........................................................................................62 

Orentas, Rimas J. ................................................................................325 

Orloff, Lisa A. .......................................................................................472 

Oro, Anthony E. ..................................................................................277 

Ortiz, Luis ...................................................................................333, 334 

Ortuno, Danny ..........................................................................339, 550 

Ortved, Kyla ...............................................................................297, 718 

Osborn, Mark J. .............................................................. 270, 584, 738 

Ostertag, Derek ..................................................................................676 

Ostrowska, Alina .......................................................... 137, 465, 470 

Otsu, Makoto .......................................................................................365 

Otus, Mokoto .......................................................................................753 

Ou, K........................................................................................................708 

Ou, Wu ....................................................................................................467 

Ousterout, David G. ................................. 167, 177, 241, 292, 491 

Owen, John ...........................................................................................253 

Owens, Jesse B. ...................................................................................569 

Oyster, Nick ..........................................................................................178 

Ozaki, Suzuka ......................................................................................192 

Ozawa, Keiya .............................................................................121, 651 

Özbas-Turan, Suna ............................................................................452 

Özkan, Naziye......................................................................................452 

Pagliara, Daria ....................................................................................300 

Pahujani, Shweta ..................................................................................66 

Pai, M. .....................................................................................................682 

Pak, Seung Hoon ................................................................................722 

Palliser, Debborah .............................................................................205 

Palmer, Donna ....................................................................................353 

Palomares, Laura A...........................................................................604 

Pan, Changchuan ...............................................................................194 

Pan, Dao............................................................................... 38, 248, 665 

Pan, Xiufang ................................................................................. 98, 166 

Pandha, Hardev ................................................................ 18, 395, 652 

Pandya, Jhell ........................................................................................333 

Pang, Li ..................................................................................................439 

Pangallo, Joey ......................................................................................673 

Pangallo, Joseph .................................................................................318 

Pangle, Amanda K. ............................................................................439 

Papapetrou, Eirini ...................................................................679, 680 

Papayannopoulou, Thalia ...................................................... 27, 427 

Pappen, Beena O. ............................199, 206, 402, 479, 503, 624 

Pâques, Frédéric ................................................................................731 

Pardi, Norbert .....................................................................................146 

Parelkar, Sangram S..........................................................................157 

Parise, Carolina B. .............................................................................658 

Park, Byung Ho ......................................................................................22 

Park, Dong-Kyu ..................................................................................598 

Park, Hana ..................................................................................497, 571 

Park, Jae .................................................................................................506 

Park, Jeonghyun .................................................................................151 

Park, Ki Dae .........................................................................................375 

Park, Sang Min ....................................................................................380 

Park, Sung Young ...............................................................................572 

Park, Woo Jin .......................................................................................380 

Park, Yeo Myeong ..............................................................................565 

Park, Yong Serk ..................................................................................159 

Parney, Ian ............................................................................................417 

Parovichnikova, E. .............................................................................724 

Partridge, Emily A. ............................................................................418 

Partridge, Terence A. ..........................................................................93 

Paruzynski, Anna ..............................................................................204 

Parvizi, Paria .......................................................................................755 

Pasceri, Peter ............................................................................322, 593 

Paschon, David E. ....................................................................107, 488 

Passineau, Michael J. ..............................................................141, 149 

Pastore, Nunzia ........................................................................353, 499 

Pastor-Flores, Ana R. .......................................................................604 

Patel, Anant B......................................................................................150 


169


Patel, Bella .................................................................................... 24, 411 

Patel, Manij ..........................................................................................535 

Patt, Richard ...........................................................................................19 

Paulard, Anais .....................................................................................674 

Paun, Marla ..........................................................................................140 

Pavani, Giulia .......................................................................................110 

Payan, Emmanual ..............................................................................674 

Payne, Kimberly J. .............................................................................468 

Peccate, Cecile ....................................................................................225 

Pei, Nana ...............................................................................................221 

Pellin, Danilo ..........................................................................................63 

Pence, Casey ........................................................................................461 

Peng, Kah Whye ....................................................................................17 

Peng, Kah-Whye ......................................................................... 20, 413 

Peng, L. ...................................................................................................682 

Peng, Rui-Yun ......................................................................................105 

Penn, Marc S. .......................................................................................500 

Pennell, Christopher A. ...................................................................270 

Perdomini, Morgane ........................................................................278 

Pereboeva, Larisa ..............................................................................587 

Perez, Giovani B. ................................................................................582 

Perez, Omar .........................................................................................703 

Perez-Pinera, Pablo ........................................... 167, 241, 292, 491 

Perkins, Edward .................................................................................657 

Perlingeiro, Rita C. R. .......................................................................168 

Perlingeiro, Rita .................................................................................169 

Perna, Serena K. .................................................................................300 

Perrigue, Patrick M. ..........................................................................656 

Persikov, Anton V. ..............................................................................485 

Persons, Derek A. ............................................................ 26, 235, 424 

Persson, Jonas ...........................................................................201, 679 

Pertschuk, Dan ...................................................................................676 

Pestina, Tamara I. ..............................................................................424 

Peterson, Jonathan ...........................................................................544 

Petrey, Aaron .......................................................................................359 

Petrov, Mihail ......................................................................................391 

Petrov, Mike ............................................................................................92 

Petry, Harald ........................................................................................274 

Pettigrew, Amy ...................................................................................461 

Phalon, Connor .............................................................. 206, 570, 633 

Phaltane, Ruhi.....................................................................................422 

Phan-Lai, Vy ............................................................................................13 

Phelan, Dane........................................................................................335 

Phillips, Dawn ........................................................................................57 

Phillips, Lucy .......................................................................................453 

Picard, Capucine ................................................................................306 

Piccolo, Pasquale .......................................................... 353, 362, 553 

Pichon, Chantal ..................................................................................342 

Piekna, Alina .......................................................................................593 

Piepenhagen, Peter A. .....................................................................453 

Pierce, Cora .............................................................................................54 

Pierciey, John ..........................................................................................37 

Pierciey, Jr, Francis H. .........................................................................51 

Pierciey, Jr, Francis J. ..............................................................251, 258 

Pierre-Destine, Lorraine ................................................................373 

Pierson, Tyler M. ................................................................................378 

Pigeon, Lucie .......................................................................................342 

Pike-Overzet, Karin ................................................................313, 319 

Pirollo, Kathleen F. ............................................................................273 

Pivirotto, Phillip ............................................................ 288, 594, 599 

Plati, Tiziana ........................................................................................309 

Plesa, Gabriela ....................................................................................508 

Plewa, Cherylene .....................................................................339, 550 

Poddar, Minakshi .....................................................................179, 611 

Podetz-Pedersen, Kelly M. ...................................................364, 495 

Podhajcer, Osvaldo L. .......................................................................635 

Podsakoff, Gregory M. .....................................................................110 

Poirot, Laurent .............................................................. 401, 683, 731 

Polach, Kevin J. ...................................................................................461 

Poliani, Pietro L. .................................................................................661 

Ponde, Datta ........................................................................................289 

Ponder, Katherine P. ............................................................................87 

Ponnazhagan, Selvarangan ...........................................................548 

Ponnusamy, Ravi ...............................................................................497 

Poppante, Kimberly ............................................................................33 

Poptani, Harish...................................................................................289 

Porada, Christopher .........................................................................253 

Portales-Casamar, Elodie ...............................................................115 

Porteus, Matthew H. ..............................................................324, 489 

Porteus, Matthew ..............................................................................471 

Posey Jr, Avery D. ...............................................................................623 

Potter, Mark ............................................................................................55 

170 



Potter, Phil ............................................................................................648 

Poulard, Karine .....................................................................................90 

Powlesland, Alex................................................................................508 

Pozsgai, Eric R. ...................................................................................172 

Prabhakar, Shilpa ..............................................................................286 

Preston, Thomas J. ...............................................................................91 

Preuss, Meredith A. ..........................................................................560 

Price, Sandra ............................................................................... 30, 428 

Prill, Jan-Michael .....................................................................126, 484 

Prince, Timothy .................................................................................660 

Prockop, Susan E. .................................................................................61 

Proto, Jonathan D. ...................................................................179, 611 

Provasi, Elena .....................................................................................504 

Psatha, Nikoleta ......................................................................... 27, 427 

Puccio, Hélène ....................................................................................278 

Pugh, John A. T. ...................................................................................158 

Pule, Martin .........................................................................................434 

Pulicherla, Nagesh ...............................................................................77 

Pulido, Jose ................................................................................395, 652 

Pumphrey, Nick J. ...................................................................... 59, 298 

Pumphrey, Nick ..................................................................................508 

Pungente, Michael D. .......................................................................755 

Qasim, Waseem ..................................................................................578 

Qi, Feng ..................................................................................................404 

Qian, Haohua .......................................................................................709 

Qiao, Chunping .............................................................. 296, 367, 370 

Qin, Hong ..............................................................................................626 

Qin, Su ....................................................................................................326 

Qiu, Jianping ........................................................................................106 

Qiu, Yujie ...............................................................................................404 

Qu, Guang .............................................................................................529 

Qu, Jinrong ...................................................................................... 50, 61 

Quach, Lucy .........................................................................................373 

Quinn, Thomas P. ...............................................................................532 

Quintanilla, Rene H...........................................................................244 

Rabinowitz, Joseph ...................................................... 108, 444, 544 

Radu, Antoneta ...................................................................................236 

Rae, Dustin T. .......................................................................................525 

Raii, Arash ...........................................................................................727 

Ragan, Regina .....................................................................................163 

Raghavarchari, Nalini .........................................................................65 

Ragni, Margaret C. .............................................................................110 

Rahimov, Fedik ......................................................................................93 

Rait, Antonina .....................................................................................273 

Raju, Liji ................................................................................................755 

Rakoczy, Elizabeth P. ...........................................................................54 

Ramos, Carlos A. ......................................................................299, 684 

Ramos, Julian N. .................................................................................543 

Ramsay, E. .............................................................................................708 

Ramya, V. ...............................................................................................114 

Ranaldo, Gabriella .............................................................................469 

Randle, A. E. ............................................................................................40 

Randle, Ashley N. ...................................................36, 377, 590, 592 

Ranghetti, Anna .................................................................................301 

Ranzani, Marco .........................................................................233, 666 

Rao, Donald ................................................................................459, 659 

Rao, Donald D. .. 199, 206, 219, 234, 272, 317, 402, 510, 625 

Rao, Nalam M. .....................................................................................156 

Rapoport, Aaron P. .......................................................... 59, 298, 508 

Rauschhuber, Christina ..................................................................557 

Rawlings, David..................................................................................673 

Rawlings, David J. ..................................................32, 311, 318, 665 

Raya, Angel ...........................................................................................469 

Razban, Vahid .....................................................................................368 

Rea-Ramsey, Angela .........................................................................461 

Reay, Daniel P. ........................................................................................94 

Rebar, Edward J. ............................................................... 46, 184, 488 

Recchia, Alessandra .........................................................................347 

Redlich, Shira ......................................................................................366 

Redmond, Eugene .............................................................................465 

Reebye, V. ..............................................................................................682 

Reed, T. D. .................................................................................................15 

Rees, Donald J. ....................................................................................616 

Reid, Michael .......................................................................................656 

Reik, Andreas ......................................................................................504 

Reinhart, Bonnie......................................................................416, 566 

Reinhart, Bonnie L. ...........................................................................478 

Reiser, Jakob ........................................................................................467 

Rejiba, Soukaina ................................................................................655 

Remenyik, Eva ....................................................................................147 

Reuser, Arnold J. J. .............................................................................275 

Reutenauer, Laurence .....................................................................278 


171


Rex, Tonia S. .........................................................................................226 

Reyon, Deepak ....................................................................................490 

Rezek, Valerie.........................................................................................48 

Rhee, Byungdoo .................................................................................151 

Rhodes, Eric ...............................................................................245, 551 

Riba, Michela .......................................................................................233 

Ribas, Antoni .......................................................................................302 

Ribeiro, Aline H. .................................................................................630 

Riberdy, Janice M. ..............................................................................235 

Rice, Jennifer .......................................................................................461 

Rice, Kevin G. ............................................................................... 41, 155 

Richard, Champlin ............................................................................431 

Richard, Isabelle ................................................. 294, 606, 610, 615 

Richie, Christopher T. ......................................................................382 

Richter, Miximilian ...................................................... 303, 483, 552 

Riddell, K. P. ............................................................................................40 

Riddell, Stan ...........................................................................................81 

Riddell, Stanley R. .............................................................................744 

Rieck, Alex ............................................................................................245 

Riggs, Arthur .......................................................................................474 

Riley, James L. .....................................................................................508 

Rinaldi, Fabrizio .......................................................................168, 169 

Riolobos, Laura .....................................................................................81 

Rissanen, Tuomas T. ............................................................................97 

Rival-Gervier, Sylvie .........................................................................322 

Rivers, Angela ...........................................................................333, 334 

Rivers, Angela E. ................................................................................542 

Rivière, Christel ............................................................................ 52, 90 

Rivière, Julie ........................................................................................662 

Riviere, Christel ....................................................................................33 

Riviere, Isabelle...............................................50, 61, 427, 506, 651 

Robbins, Joan M. ................................................................................676 

Robbins, Joan ......................................................................................703 

Robbins, Paul D. ....................................................................................94 

Roberts, Alison ...................................................................................678 

Roberts, Errin .....................................................................................453 

Roberts, Sean ......................................................................................681 

Roberts, Thomas C. ...........................................................................181 

Robison, Richard ...............................................................................208 

Rocca, Celine J. ............................................................... 493, 494, 546 

Rocca, Céline .......................................................................................678 

Roche, Francis P. ................................................................................631 

Rockey, William M. ............................................................................509 

Rockne, Russell C. ................................................................................25 

Rodino-Klapac, Louise R. ............................................. 91, 172, 291 

Rödl, Wolfgang ...................................................................................134 

Rodriguez, Stephen ..........................................................................263 

Roh, Jae-Kyu ................................................................... 143, 598, 602 

Romain, Gabrielle ..............................................................................393 

Romano, Josephine ..............................................................................46 

Romero, Zulema ....................................................................................26 

Rommelfanger-Konkol, Diana ...........................................505, 652 

Roncarolo, Maria Grazia .......................................................323, 441 

Rondon, Gabriela ..................................................................................10 

Rooney, Cliona M. ..............................14, 299, 396, 398, 400, 622 

Roovers, Onno ....................................................................................275 

Rosales, Cecilia .........................................................................363, 580 

Rose, Laura ................................................................................161, 162 

Rose-Hellkant, Teresa .....................................................................657 

Rosenberg, Jonathan B. .........................................................373, 589 

Rosenberg, Steven A. .......................................................................716 

Rosengurt, Enrique ..........................................................................659 

Ross, Joel ...............................................................................................593 

Rossi, John J. ............................................... 454, 456, 515, 668, 682 

Rossini, Silvano ..................................................................................420 

Roth, David B.......................................................................................324 

Roth, Justin C. ......................................................................................438 

Rothe, Michael .................................................................. 37, 422, 425 

Rottier, Peter .......................................................................................707 

Roudaut, Carinne ....................................................................294, 615 

Rouillon, Jérémy ................................................................................612 

Round, June L. .....................................................................................712 

Rousseau, Joel .....................................................................................293 

Rousseau, Joël ............................................................................. 83, 389 

Rowland, Elizabeth F. ......................................................................485 

Roy, Andre ............................................................................................495 

Roy, Aparna ................................................................................474, 476 

Roy-Chaudhuri, Biswajoy ..............................................................436 

Roy-Chaudhuri, Biswajoy ..............................................................701 

Rozengurt, Nora .................................................................................497 

Ruan, Zhechao ....................................................................................607 

Rubin, David G. ...................................................................................589 

172 



Ruggiero, Eliana ......................................................................... 47, 204 

Ruiz, Autumn ......................................................................................407 

Rungta, R. ..............................................................................................708 

Runtsch, Marah C. .............................................................................712 

Ruozi, Giulia ................................................................................. 95, 368 

Rushworth, David .............................................................................202 

Russ, Jill .................................................................................................524 

Russell, David W......................................................................... 81, 266 

Russell, Rob .........................................................................................516 

Russell, Stephen J. ..........................................17, 20, 406, 407, 413 

Russo, Fabio .........................................................................................441 

Rusten, Myra C. ..................................................................................102 

Ryals, Renee C. .......................................................................................55 

Ryan, Robin .............................................................................................14 

Ryu, Byoung ................................................................................. 37, 674 

Ryu, Byoung Y. .......................................................................................51 

Sabapathy, Vikram ............................................................................754 

Sabine, Geiger-Schredelseker ......................................................307 

Sacher, Anna ........................................................................................123 

Sadelain, Michel ................................... 50, 61, 427, 506, 651, 728 

Sætrom, P. .............................................................................................682 

Sagara, Miyako ............................................................................ 69, 440 

Sagawa, Hiroaki .................................................................................532 

Sahel, José-Alain ................................................................................225 

Sahenk, Zarife .....................................................................................171 

Saito, Hiroki ...............................................................................192, 654 

Sakamaki, Ippei ..................................................................................626 

Sakamoto, Chika ........................................................... 432, 440, 442 

Sakata, Asuka ......................................................................................121 

Sakata, Yoichi ............................................................................121, 237 

Sakuma, Toshie .......................................................................... 68, 726 

Sakurai, Fuminori .............................................................................482 

Sakurai, Hidetoshi .............................................................................715 

Salazar, Andres M. .............................................................................270 

Salibi, N. ....................................................................................................40 

Salmon, Florence ...............................................................................274 

Salomon, Daniel R. ............................................................................493 

Salva, Emine ........................................................................................452 

Samara-Kuko, Ermira ......................................................................355 

Samaranch, Lluis .......................................................... 288, 594, 599 

Samoto, Vivian Y. ...............................................................................582 

Samulski, R. Jude .......................................6, 54, 74, 112, 268, 546 

San Sebastian, Waldy .................................................. 288, 594, 599 

Sanadiki, Sara .....................................................................................492 

Sanchez, Robbi ...................................................................................272 

Sandikin, Daren ..................................................................................430 

Sandoval, Lucia ........................................................................152, 620 

Sandoval-Zamora, Hugo .......................................................246, 564 

Sandoval-Zamora, Hugo E. ............................................................534 

Santiago, Yolanda ..............................................................................568 

Sanvito, Francesca ....................................................... 233, 323, 666 

Saori, Ukai ............................................................................................142 

Sapp, Peter ...........................................................................................603 

Sardesai, Niranjan Y. ....................................... 9, 45, 153, 341, 397 

Sardi, Pablo S..........................................................................................34 

Sargent, R. G. .......................................................................................472 

Sargent, Roy G. .........................................................................471, 575 

Sarkar, Debalina .................................................................................109 

Sartin, J. L. ................................................................................................40 

Sasakawa, Noriko ..............................................................................715 

Sather, Blythe D. ....................................................................................32 

Sato, Makoto ........................................................................................215 

Sato, Taku .............................................................................................753 

Savchenko, V. .......................................................................................724 

Savola, Paula ........................................................................................730 

Savoldo, Barbara .....................49, 197, 299, 300, 305, 392, 394 

Sawaguchi, Yoshikazu .....................................................................145 

Saydaminova, Kamola ................................................ 679, 680, 704 

Sayles, Leanne C. ................................................................................436 

Scala, Serena ...........................................................................................28 

Scaramuzza, Samantha ......................................................................63 

Scaria, Puthupparampil ..................................................................455 

Schaffer, David V. .................................................3, 5, 267, 327, 692 

Schambach, Axel .......................................................66, 84, 313, 425 

Scharenberg, Andrew M. .............188, 311, 318, 401, 673, 683 

Schech, Amanda .................................................................................636 

Scheel, Maria .......................................................................................283 

Scheff, Nicole .......................................................................................285 

Scherer, Lisa.........................................................................................668 

Schiedlmeier, Bernd ...............................................................320, 429 

Schiffer-Mannioui, Cécile ...............................................................401 

Schinco, Piercarla ..............................................................................469 


173


Schiroli, Giulia .....................................................................................486 

Schlichting, Lisa .................................................................................497 

Schmidt, Manfred ...........................37, 47, 62, 63, 204, 210, 233, 

......................................................274, 279, 309, 518, 666, 678, 738 

Schmidt, Manuel ................................................................................686 

Schmidt, Sabine .................................................................................274 

Schneider, Lynda ...............................................................................721 

Schneppendahl, Johannes .............................................................178 

Schoield, Christine ..........................................................................551 

Scholler, John .......................................................................................623 

Schroff, Matthias ................................................................................686 

Schubert, Kathryn .............................................................................466 

Schuermann, Nina ............................................................................516 

Schug, Jonathan .................................................................................581 

Schuldt, Ryan A. .................................................................................745 

Schwäble, Joachim ...............................................................................66 

Schwerk, Nicolaus ................................................................................84 

Scripps, Vicky .........................................................................................56 

Seabra, Miguel C. ...............................................................................227 

Sekaly, Raick-Pierre ...........................................................................58 

Selby, Peter ......................................................................... 18, 395, 652 

Sellers, Stephanie E. ............................................................................65 

Selot, Ruchita ......................................................................................549 

Semechkin, Ruslan ....................................................... 137, 465, 470 

Semenova, Ekaterina .......................................................................578 

Sen, Dwaipayan ......................................... 114, 331, 337, 536, 537 

Senac, Julien S. ....................................................................................280 

Sena-Esteves, Miguel ...................... 1, 36, 40, 93, 212, 286, 377, 

.......................................................384,387, 388, 590, 592, 595, 721 

Senzer, Neil ............................199, 206, 219, 234, 272, 273, 317, 

............................................459, 479, 503, 510, 570, 624, 625, 659 

Seo, Emiko ............................................................................................643 

Seog, Joonil ..............................................................................................44 

Sergi Sergi, Lucia ...................................................64, 486, 666, 667 

Sergio, Bossi ........................................................................................520 

Serrano-Pozo, Alberto .....................................................................540 

Servais, Laurent .................................................................................734 

Sessa, Maria .........................................................................................309 

Seth, Prem ............................................................................................555 

Seto, Jane T. ..........................................................................................543 

Sevin, Caroline ....................................................................................589 

Sganga, Leonardo ..............................................................................635 

Sgouramali, Eleni ..............................................................................427 

Shaban, Mona......................................................................................451 

Shah, Riddhi ........................................................................................345 

Shah, Sapana N. ..................................................................................451 

Shanley, Ryan ......................................................................................414 

Shapiro, Michael ...................................................................................44 

Sharma, Anurag .......................................................................104, 628 

Sharma, Jay ................................................................................493, 494 

Sharma, Nitasha .................................................................................288 

Sharma, Priyanka ..............................................................................267 

Sharma, Rajiv ......................................................................................488 

Sharon, David ......................................................................................403 

Shaw, Kit L. ...........................................................................................307 

Shayakhmetov, Dmitry M. ..............................................................477 

Shedlock, Devon J. ................................................................................45 

Shelke, Rajani ......................................................................................330 

Shen, Kang ............................................................................................255 

Shen, Shen ....................................................................................... 73, 77 

Shi, Gang................................................................................................361 

Shi, Hailiang .........................................................................................561 

Shi, Yijun ...............................................................................................378 

Shi, Yingai .............................................................................................473 

Shields, Jordan E. ...............................................................................663 

Shigemura, Katsumi .........................................................................192 

Shihabuddin, Lamya S. ............................................................ 34, 282 

Shiku, Hiroshi ............................................................................. 16, 629 

Shim, Inkyong .....................................................................................237 

Shima, Midori ......................................................................................357 

Shimada, Hideaki ..............................................................................216 

Shimada, Takashi .......................................................... 228, 358, 601 

Shimada, Yohta .........................................................................365, 753 

Shimada, Yota......................................................................................585 

Shimizu, Hiroyuki ..................................................................... 69, 440 

Shimizu, Kahori ..................................................................................482 

Shimizu, Saki .......................................................................................530 

Shin, Bae-Hyun ...................................................................................380 

Shin, Jeong-A. ......................................................................................238 

Shin, Jin-Hong ................................................................... 98, 166, 613 

Shin, Simon C. ...........................................................................665, 670 

Shin, Sug Kyun ....................................................................................685 

Shindler, K. ..............................................................................................53 

174 



Shirakawa, Toshiro .................................................................192, 654 

Shires, G. Thomas ..............................................................................402 

Shirley, Shawna .....................................................................................99 

Shirosaki, Chihiro ..............................................................................192 

Shiver, Rachel .........................................................................................45 

Shoemark, Amelia .............................................................................435 

Shontz, Kim .............................................................................................91 

Shontz, Kimberly M. .........................................................................291 

Shpall, Elizabeth J. ................................................................................10 

Shpall, Elizabeth ................................................................................650 

Shreiber, Claire A. ..............................................................................726 

Shum, Kato ...........................................................................................515 

Siametis, Athanasios ........................................................................427 

Sidman, Richard L. ...............................................................................34 

Silva, Anne ............................................................................................744 

Silva, Gabriela .....................................................................................160 

Silva, Michael E. ..................................................................................656 

Silva, Nilsa ............................................................................................387 

Silverman, Lara I. ...............................................................................619 

Silvin, Christopher ............................................................................307 

Simoneau, Mélanie ...........................................................................135 

Simpson, Elizabeth M. .....................................................................115 

Siner, Joshua I. ....................................................................................498 

Singh, Aruna ........................................................................................576 

Singh, Harjeet .....................................10, 393, 502, 621, 649, 650 

Singh, Mandeep S. .............................................................................229 

Singh, Martin .......................................................................................639 

Singh, Mona .........................................................................................485 

Singh, Purba ........................................................................................200 

Singh, Swati .........................................................................................673 

Singha, Swati ..........................................................................................32 

Skinner, Nicole E. B. ..........................................................................270 

Skog, Johan .................................................................................304, 751 

Skorik, Christian ......................................................................260, 262 

Skottman, H .........................................................................................732 

Slavcev, Roderick .....................................................................688, 713 

Slepushkin, Vladimir .......................................................................495 

S-Manickam, Devika .........................................................................691 

Smethurst, Dominic P. ............................................................. 59, 298 

Smirnova, Olga ...................................................................................581 

Smith, Andrew .......................................................................................88 

Smith, Barbara K. ...................................................................... 57, 617 

Smith, Barbara .......................................................................................90 

Smith, Bruce F. ....................................................................................613 

Smith, Catherine ................................................................................284 

Smith, Claire M. ..................................................................................435 

Smith, Julianne ...................................................................................401 

Smith, Julianne ...................................................................................683 

Smith, Krissy .......................................................................................660 

Smith, Trevor R. F. .............................................................................153 

Smogorzewska, Monika..................................................................307 

Snyder, Evan ........................................................................................465 

Snyder, Richard O. ........................................................................ 60, 76 

Soderini, Alejandro ...........................................................................635 

Soheili, Tayebeh .................................................................................662 

Sohn, Jihee ............................................................................................250 

Soker, Shay ...........................................................................................253 

Soker, Thomas .......................................................................................33 

Sokolic, Robert ...................................................................................307 

Soland, Melisa .....................................................................................253 

Sommerville, Laura ............................................................................... 2 

Sondhi, Dolan .................................................................... 11, 373, 589 

Song, Lina .............................................................................................603 

Song, Lina .............................................................................................735 

Song, Liujiang............................................................................328, 542 

Song, Shuxian ............................................................................101, 140 

Song, Sihong ........................................................................................545 

Song, Xiao-Tong ..................................................................................410 

Soo, Shin Back .....................................................................................699 

Sorrentino, Brian P. ..................................................... 308, 527, 671 

Sortwell, C. E. ......................................................................................381 

Sortwell, Caryl E. .....................................................................386, 596 

Sosale, Nisha G. ..................................................................................523 

South, Andy P. .....................................................................................578 

Sowers, Anastasia .............................................................................743 

Sparks, Jeff............................................................................................461 

Spencer, H. Trent ............................................................. 31, 528, 663 

Spencer, Jacqueline F. ......................................................................480 

Spillman, Michael ..............................................................................336 

Spires-Jones, Tara .............................................................................283 

Spitzweg, Christine ...........................................................................134 

Springer, Matthew L. ........................................................................369 


175


Squadrito, Mario L. ...........................................................................301 

Sridharan, Arati M. ...........................................................................100 

Srinivasan, N..............................................................................114, 536 

Srivastava, Alok ......................................... 114, 331, 337, 536, 537 

Srivastava, Arun ....................................................75, 118, 314, 328, 

......................................................331, 333, 334, 541, 542, 545, 613 

Staal, Frank J. T. ........................................................................313, 319 

Stadtmauer, Edward A. ................................................. 59, 298, 508 

Stagg, Scott ...........................................................................................336 

Stamatoyannopoulos, George .............................................. 27, 427 

Stanek, Lisa M. ....................................................................................282 

Stanic, Bojana .....................................................................................511 

Starker, Colby G. .......................................................................584, 738 

Starnes, Joseph ...................................................................................664 

Steckler, Robert ........................................................................199, 624 

Stedman, Hansell ....................................................................... 92, 391 

Steet, Richard ......................................................................................359 

Stefankski, Jolanta ............................................................................506 

Stefano, Corna.....................................................................................520 

Stein, Libby ..........................................................................................492 

Stein, Sarah ................................................................................152, 620 

Steines, Benjamin R..........................................................................327 

Stewart, Phoebe L. ............................................................................477 

Stilhano, Roberta S. ..................................................... 265, 582, 658 

Stock, Stuart ........................................................................................555 

Stockard, Cecil R. ...............................................................................560 

Stoddard, Barry ........................................................................188, 318 

Stoddard, Barry L. .............................................................................193 

Stoica, Lorelei I. ..................................................................................388 

Stojdl, David F. ....................................................................................413 

Stok, Merel ...........................................................................................275 

Stoltzman, Carrie ...............................................................................450 

Stolz, Donna B. .......................................................................................94 

Stone, Daniel .................................................................. 187, 188, 193 

Storb, Rainer .......................................................................................443 

Stout, David .........................................................................................497 

Stoytchev, Ilko .....................................................................................569 

Strange, Wanda ..................................................................................351 

Strauss, Bryan E. ......................................................................519, 630 

Streeter, Jennifer ................................................................................511 

Streetz, Konrad...................................................................................514 

Stringer, C. Allen ................................................................................503 

Stripecke, Renata .................................................................................47 

Stupka, Elia ................................................................................233, 517 

Su, Leonard .............................................................................................92 

Su, Qin ........................................................................................4, 93, 512 

Su, Quin .................................................................................................384 

Su, Rui-Jun ............................................................................................468 

Su, Xiaomin ..........................................................................................457 

Subrizi, A ...............................................................................................732 

Sudha, G. ................................................................................................536 

Sudha, Govindarajan ........................................................................114 

Suel, Laurence ....................................................................................294 

Suel-Petat, Laurence ........................................................................615 

Suerbaum, Sebastian .......................................................................425 

Suerth, Julia D. ............................................................................ 66, 425 

Sugimoto, Mitsuhiko ........................................................................357 

Sui, Hongshu .......................................................................................535 

Suksanpaisan, Lukkana .....................................................................20 

Sullivan, Lisa ........................................................................................224 

Sumaroka, Marina V. ........................................................................374 

Sumaroka, Marina .............................................................................547 

Summers, Alex ....................................................................................119 

Sun, Baodong ......................................................................................276 

Sun, Helena C. .....................................................................................107 

Sun, Jiali ....................................................................................................14 

Sun, Ryan R. .........................................................................................140 

Sun, Ryan ..............................................................................................101 

Sun, Xiao-Xin ..........................................................................................72 

Sun, Xingshen .....................................................................................535 

Sun, Yu ...................................................................................................550 

Sung, Biin ..............................................................................................104 

Suppa, Erica .........................................................................................298 

Surace, Enrico Maria ........................................................................539 

Sutton, Debbie ....................................................................................508 

Su-Youne, Han .....................................................................................699 

Suzuki, Daichi .....................................................................................142 

Suzuki, Ryo .................................................................................142, 145 

Suzuki, Shingo ............................................................... 471, 472, 575 

Svinartchouk, Fedor ...............................................................445, 612 

Swanekamp, Ria L. ...............................................................................43 

Swanson, Kristin R. ..............................................................................25 

176 



Swanson, Magdalena I. ....................................................................115 

Swaroop, Anand .................................................................................709 

Sweeney, Colin L. ..................................................................................82 

Sweet-Cordero, E. A. .........................................................................436 

Swiderski, P. .........................................................................................682 

Switzer, Kirsten C. .............................................................................502 

Switzer, Kirsten ............................................................. 198, 621, 649 

Symonds, Geoff P. .................................................................................48 

Synold, Timothy W. ...........................................................................648 

Sysol, Justin R. .....................................................................................280 

‘t Hoen, Peter A. .................................................................................609 

Tabar, Vivianne S. .................................................................................11 

Tabor, Aaron T. ....................................................................................616 

Tac, Kim Jin ..........................................................................................699 

Tachibana, Masashi ..........................................................................482 

Tada, Yuji ...............................................................................................216 

Taga, Yavuz ...........................................................................................697 

Tagalakis, Aristides D. ...........................................................158, 434 

Tagawa, Masatoshi ....................................................... 216, 638, 642 

Taherian, Mehran ..............................................................................286 

Tai, Alex .................................................................................................110 

Tai, Chun-Hwei ......................................................................................60 

Tai, Ming-Hong ...................................................................................230 

Tai, Ming-Hong ...................................................................................740 

Takafuji, Makoto ................................................................................150 

Takagi, Kazuko ...................................................................................627 

Takagi, Norio .......................................................................................142 

Takemasa, Ichiro................................................................................749 

Takagi-Kimura, Misato ..........................................................218, 638 

Takahashi, Hiroshi ............................................................................228 

Takahashi, Masamichi ............................................................. 70, 522 

Takapoo, Maysam ..............................................................................511 

Takayama, Koichi...................................................69, 432, 440, 442 

Takayama, Koji ...................................................................................614 

Takeda, Shin’ichi ........................................................... 120, 176, 608 

Takeda, Shuko .....................................................................................283 

Takesako, Kazuto ...............................................................................629 

Takesako, Kazutoh ............................................................................651 

Takeuchi, Ryo ......................................................................................193 

Takiguchi, Yuichi ................................................................................216 

Talbot, Kendra T. ...................................................................................45 

Talmon, Maria .....................................................................................469 

Tam, Hok-Hei.......................................................................................412 

Tam, Quincy .........................................................................................522 

Tam, Y. ....................................................................................................708 

Tamai, Katsuto .......................................................................................80 

Tamamoto, Atsuko ............................................................................638 

Tan, Jian .................................................................................................178 

Tan, Lay Geok ......................................................................................430 

Tan, Mengqun .....................................................................................328 

Tan, Menqun ........................................................................................542 

Tandeski, Terry ..................................................................................619 

Tandon, Manish ..................................................................................628 

Tang, Qiushi .........................................................................................211 

Tang, Qiushi .........................................................................................735 

Tang, Ru-Hang ....................................................................................367 

Tang, Terence ......................................................................................214 

Tang, Winson .........................................................................................58 

Tang, Ying ..........................................178, 179, 250, 281, 290, 611 

Tani, Kenzaburo ..............................................69, 86, 432, 440, 442 

Tannous, Bakhos A. ................................. 103, 321, 458, 540, 751 

Tapscott, Stephen H. ........................................................................443 

Taquet, Nicolas .............................................................. 352, 464, 633 

Tarantal, Alice F. .................................................................................542 

Targovnik, Alexandra M. ................................................................632 

Tatsumi, Kohei ............................................................................ 79, 237 

Tatsumi, Koichiro ..............................................................................216 

Taylor, Catherine................................................................................214 

Taylor, Clare ............................................................................................61 

Taylor, J. .................................................................................................708 

Taylor, Walton ...........................................................................199, 624 

Tayton-Martin, Helen K. .................................................................298 

Teckman, Jeffery ................................................................................211 

Templeton, Nancy S. .................................................... 464, 570, 625 

Tengood, Jillian E. ..............................................................................144 

Tenhu, H ................................................................................................732 

Tenney, Rebecca .................................................................................444 

Terada, Naohiro .................................................................................545 

Terjung, Ronald L. .............................................................................166 

Tesfay, Mulu Z. ....................................................................................413 

Teunissen, Erik ...................................................................................707 

Thakore, Pratiksha I. ............................... 167, 170, 177, 292, 491 


177


Thakur, Archana, ...............................................................................711 

Tharion, George .................................................................................754 

Tharmalingam, Sujeenthar ...........................................................390 

Themeli, Maria ...................................................................................728 

Theobald, Narda ................................................................................308 

Thiel, William H. ................................................................................511 

Thokala, Radhika .......................................................... 431, 502, 649 

Thomas, Amelia H. ...............................................................................51 

Thomas, Carole ..................................................................................185 

Thompson, David H. .........................................................................745 

Thompson, Jill .........................................................18, 395, 505, 652 

Thompson, John .................................................................................214 

Thony, Beat ..........................................................................................354 

Thorgeirsson, Snorri S. ...................................................................217 

Thorpe, Robin .....................................................................................526 

Thrasher, Adrian J. ................................... 306, 313, 578, 661, 678 

Throm, Robert E. ...............................................................................672 

Thyme, Summer ................................................................................318 

Ti juan, Mi .............................................................................................649 

Tian, Gengwen .......................................................................................12 

Tian, Jie ..................................................................................................133 

Tiberghien, Pierre .............................................................................399 

Tisdale, John ................................................................................ 30, 428 

Tisdale, John F. .......................................................................................65 

Tobin, Lisa A. .......................................................................................513 

Todorow, Carlyn A.............................................................................236 

Tolar, Jakub ........................................................................ 62, 584, 738 

Tolmachova, Tanya ...........................................................................227 

Tomalia, D.A. ........................................................................................682 

Tomita, Kyoko .....................................................................................482 

Tompkins, Josh ...................................................................................474 

Tonon, Giovanni .................................................................................233 

Torbett, Bruce ........................................................................................32 

Torgerson, Troy ..................................................................................673 

Torgerson, Troy R. ............................................................................311 

Toro, Gabriela .....................................................................................603 

Toro, Gabriella ....................................................................................388 

Torok-Storb, Beverly ..............................................................419, 423 

Torrance, Chris ...................................................................................551 

Torres-Vega, Miguel A. .....................................................................604 

Torry, Donald S. ..................................................................................200 

Toso, Erik ..............................................................................................169 

Toth, Karoly .........................................................................................480 

Tournaire, Benoit .................................................................................76 

Towe, Christopher ............................................................................438 

Trabalza, Antonio .................................................................................67 

Trabucco, Leonardo .........................................................................516 

Tran, Chy-Anh .....................................................................................668 

Tran, Dat ...............................................................................................431 

Treger, Janet ..............................................................................522, 530 

Tremblay, Jacques P. ...................................83, 293, 389, 491, 620 

Tricoli, Lucas ..........................................................................................44 

Triggs-Raine, Barbara .....................................................................379 

Trobridge, Grant ................................................................................673 

Trobridge, Grant D. .................................................................311, 525 

Troupes, Andrew ..................................................................................77 

Trump, Lisa ..........................................................................................426 

Truong, Lynn .......................................................................................183 

Tsai, Hsin-Yin.......................................................................................545 

Tsai, Mu-Rou ........................................................................................723 

Tsai, Richard K. ...................................................................................523 

Tsai, Shengdar Q. ...............................................................................490 

Tschudy-Seney, Benjamin ..............................................................468 

Tseng, Sheng-Hong ..............................................................................60 

Tsui, Christopher ...............................................................................302 

Tsuji, Kohichiro .....................................................................................86 

Tsujita, Maristela ...............................................................................519 

Tsukahara, Tomonori ............................................................121, 651 

Tuan, Rocky .........................................................................................178 

Turchiano, Giandomenico .............................................................347 

Turner, Lorianne S. ...........................................................................660 

Turner, Mallory A. ...................................................................132, 481 

Turtle, Cameron J. .............................................................................744 

Turtle, Cameron ....................................................................................81 

Turunen, Heikki T..............................................................................330 

Tzen, Kai-Yuan .......................................................................................60 

Uchibori, Ryosuke ...................................................................121, 651 

Uchida, Naoya ......................................................................30, 65, 428 

Uchino, Junji ........................................................................................558 

Udd, Bjarne ..........................................................................................615 

Uemura, Mamoru ..............................................................................749 

Ueno, Kazuko .........................................................................................86 

178 



Ugai, Hideyo...............................................................................127, 558 

Ugen, Kenneth E. .................................................................................... 9 

Uitto, Jouni ..............................................................................................80 

Ukovich, Laura ....................................................................................368 

Ul-Hasan, Tahira ................................................................................542 

Uludag, Hasan ...................................................... 161, 162, 213, 462 

Ungerechts, Guy ............................................................ 405, 554, 639 

Ur, Sarah ......................................................................................493, 494 

Ur, Sarah M. ..........................................................................................546 

Urabe, Masashi .........................................................................121, 651 

Urbinati, Fabrizia .................................................................................26 

Uribe, Misael .......................................................................................604 

Urnov, Fyodor D. ................................................................................568 

Ürenden, Gökçe ..................................................................................204 

Urschitz, Johann.................................................................................569 

Urtti, A ....................................................................................................732 

Usas, Arvydas ............................................................................178, 290 

Ustoa, Maria.........................................................................................534 

Utoh, Rie ...................................................................................................79 

Vázquez-Carcaño, Lizbeth M. .......................................................604 

Vähä-Koskela, Markus .....................................................................501 

Valdmanis, Paul N. ............................................................................436 

Valles-Ayoub, Yadira ..............................................................152, 620 

Van de Graaf, Benjamin ........................................................373, 589 

van der Bom, Imramsjah M. J. ......................................................387 

van der Horst, Gijsbertus T. J. .......................................................147 

van der Ploeg, Ans T. ........................................................................275 

van der Velden, Guus .......................................................................661 

van der Vos, Kristan E. ....................................................................304 

van Dongen, Jacques J. M. ..............................................................313 

van Helsdingen, Yvette ....................................................................521 

Van Hoppe, Stephanie .....................................................................458 

van Kalle, Christof ................................................................................62 

van Logtenstein, Richard ...............................................................274 

van Ommen, Gert-Jan B. .................................................................609 

van Putten, Maaike ...........................................................................609 

van Rijn, Sjoerd ..................................................................................321 

van Til, Nico P. .....................................................................................275 

van Til, Niek P............................................................................521, 661 

Van Vliet, Kim .................................................... 4, 71, 333, 334, 541 

Van Wittenberghe, Laetitia ...............................................33, 76, 90 

Vandenberghe, Luk H. ............................................................. 71, 330 

VandenDriessche, Thierry ..........................8, 354, 355, 496, 667 

Vandertop, W. Peter ..........................................................................321 

Varadarajan, K. S. ...............................................................................150 

Varadarajan, Navin ...........................................................................393 

Varela-Rohena, Angel ......................................................................508 

Varga, Monika .....................................................................................369 

Vargas-Jerónimo, Roxana Y. ..........................................................604 

Vasireddy, Vidyullatha ..........................................................224, 227 

Vatakis, Dimitrios N. ...........................................................................48 

Vazin, Tandis ............................................................................................ 3 

vemula, Sai vikram V. .......................................................................628 

Venditti, Charles P. ..................................................................113, 280 

Vera, Juan ..............................................................................................400 

Vera, Juan F. ................................................................................398, 622 

Veres, Gabor .....................................................37, 51, 251, 258, 674 

Verga, Michael ....................................................................................124 

Vergara-Silva, A. ....................................................................................15 

VerHeul, Ross ......................................................................................745 

Verhoeyen, Els ....................................................................................662 

Vetrini, Francesco .............................................................................353 

Vetter, Alexandra ...............................................................................134 

Vickers, Selwyn M. ............................................................................414 

Viecelli, Hiu Man ................................................................................354 

Vignaud, Alban ................................................33, 90, 240, 605, 606 

Vile, Richard ............................................................18, 395, 505, 652 

Villa, Anna ............................................................................................661 

Villamizar, Olga ..................................................................................235 

Villaverde, Marcela S. ......................................................................632 

Vincent, Karen ....................................................................................338 

Vincent, Phil ........................................................................................264 

Virdi, Kulpreet S. ................................................................................134 

Visel, Meike ............................................................................................... 5 

Visser, Trudi P. ....................................................................................275 

Vite, Charles H. ...................................................................................289 

Vitek, Libor ..........................................................................................353 

Vizcaino-Dorado, Pablo ..................................................................534 

Vlahu, Alexander ...............................................................................745 

Vogelbaum, Michael A. ....................................................................676 

Vogl, Dan T. ...........................................................................................298 

Voit, Thomas .............................................................................225, 734 


179


Voit, Tomas .................................................................................445, 612 

Volpin, Monica ............................................................................ 64, 666 

von Kalle, Christof ......................................63, 204, 233, 274, 279, 

.............................................309, 405,518, 639, 666, 674, 678, 738 

Vossough, A. ............................................................................................53 

Voytas, Daniel F. .......................................................................584, 738 

Voziyanov, Yuri ...................................................................................345 

Voziyanova, Eugenia ........................................................................345 

Vrecenak, Jesse ...................................................................................418 

Vrecenak, Jesse D. ..............................................................................236 

Vuidepot, Annelise ............................................................................508 

Vulin, Adeline ......................................................................................180 

Wack, Katy ...............................................................................................94 

Waddington, Simon ..................................................... 363, 430, 580 

Wadsworth, Sam................................................................................338 

Wagemaker, Gerard ..................................................... 275, 521, 661 

Wagner, C. R. ........................................................................................710 

Wagner, Ernst .....................................................................................134 

Wagner, John E. ..................................................................................738 

Wagner, Kathryn R. ..............................................................................93 

Wake, Kenjiro .........................................................................................79 

Walia, Jagdeep S. ................................................................................379 

Walker, Christopher M. ......................................................................91 

Walker, Edith .......................................................................................195 

Walker, Timothy.................................................................................284 

Wallace, Kirby .....................................................................................461 

Wallace, Lindsay ......................................................................171, 295 

Wallace, Lindsay M. ..........................................................................182 

Wallraven, Gladice ...............199, 203, 206, 351, 402, 577, 625 

Walsh, C. ................................................................................................708 

Walters, Jewell N. ..............................................................................397 

Wambua, Gerald ................................................................................465 

Wanatabe, Ayumi ..............................................................................432 

Wang, Beibei ...........................................................................................69 

Wang, Bing ........................................178, 179, 250, 281, 290, 611 

Wang, Chi-Hsien ......................................................................296, 370 

Wang, Dan ................................................................................................. 4 

Wang, Gensheng ................................................................................106 

Wang, Hongjie ...........................................................................483, 552 

Wang, Hongmei ..................................................................................308 

Wang, Hua ..................................................................................105, 644 

Wang, Jianbin ................................................46, 107, 183, 184, 487 

Wang, Jinhui ........................................................................................544 

Wang, Jiwei ..........................................................................................507 

Wang, Juan ...........................................................................................457 

Wang, Lan ................................................................................................11 

Wang, Lina .......................................................................... 75, 118, 314 

Wang, Lisa ............................................................................................400 

Wang, Li-Sheng .........................................................................105, 644 

Wang, Minghui....................................................................................127 

Wang, Ningqing ..................................................................................154 

Wang, Pengju.............................................................................507, 561 

Wang, Ping ...........................................................................................360 

Wang, Pin-Yi.........................................................................................412 

Wang, Qingtao ....................................................................................194 

Wang, Shelley .........................................................................................58 

Wang, Shengyao .................................................................................563 

Wang, Sheri ..........................................................................................281 

Wang, Weijun ......................................................................................703 

Wang, Xi.................................................................................................163 

Wang, Xiang .........................................................................................562 

Wang, Xiaofei ......................................................................................512 

Wang, Xiaoyin .....................................................................................369 

Wang, Xin ..............................................................................................752 

Wang, Xiuyan .......................................................................50, 61, 506 

Wang, Xuefeng ....................................................................................665 

Wang, Xueqian .......................................................................................32 

Wang, Yaohe ..............................................................................507, 561 

Wang, Yuan ..............................................................75, 118, 314, 328 

Wang, Yuhan ................................................................................ 85, 579 

Wang, Yupeng .....................................................................................318 

Wang, Zejing ........................................................................................443 

Wang, Zhaohui .................................206, 219, 234, 317, 510, 625 

Wang, Zhimin ............................................................................141, 149 

Ware, Tierra................................................................................. 39, 597 

Warischalk, Jayme K. ............................................................................ 6 

Wasielewska, Teresa ...........................................................................50 

Wasserman, Wyeth W. .....................................................................115 

Watanabe, Natsumi ..........................................................................237 

Watanabe, Norihiro ................................................................398, 622 

Watson, Rachael S. ...............................................................................88 

Watts, Ashlee E. .....................................................................................89 

180 



Watts, Korashon L. ............................................................................249 

Wayne, Alan .........................................................................................307 

Wayne, Alan S. ....................................................................................325 

Weagel, Evita .......................................................................................208 

Weaver, Eric .........................................................................................583 

Weaver, Matthew S. .............................................................................96 

Weber, Gerrit .......................................................................................392 

Weber, Nicholas D. ............................................................................193 

Weber, Nick ................................................................................187, 188 

Webber, Beau R. .................................................................................738 

Weeden, Timothy ..............................................................................453 

Wei, Dong ..............................................................................................281 

Wei, Jie ......................................................................................................12 

Wei, Lisa L. ...........................................................................................737 

Wein, Nicolas .......................................................................................180 

Weinberg, Marc S. .............................................................................189 

Weiner, David B. ................................................ 9, 45, 341, 374, 397 

Weinstein, John ..................................................................................267 

Weismann, Cara M. .................................................................384, 595 

Weiss, Brendan...................................................................................298 

Weissman, Drew ......................................................................146, 147 

Weissman, Irving ..................................................................................29 

Weitzel, Richard .................................................................................428 

Welch, Peter .........................................................................................706 

Wellman, J. ...............................................................................................53 

Wendland, Rebecca ..........................................................................104 

Wengerter, Brian C. ...........................................................................205 

Wentworth, Bruce M. .......................................................................453 

Werling, Natalie .................................................................................526 

Wernersson, Karin ............................................................................209 

Westin, Erik .........................................................................................587 

Westmoreland, Susan ......................................................................387 

Wetzke, Martin ......................................................................................84 

Wherley, Jennifer ..................................................................................26 

Whisenant, Thomas .........................................................................493 

Whitaker, Leslie R. ............................................................................382 

White, Edward A. ...............................................................................158 

Whitlock, B. K.........................................................................................40 

Wiekmeijer, Anna-Sophia ....................................................313, 319 

Wielgosz, Matthew M. .....................................................................672 

Wierda, William G. ............................................................................502 

Wilber, Andrew ................................................................ 26, 200, 235 

Wild, A ....................................................................................................708 

Wilkinson, Leslie ...............................................................................461 

Willems, Jessica ............................................................................ 8, 496 

Wille-Reece, Ulrike ..............................................................................45 

Williams, Daniel .................................................................................298 

Williams, James ..................................................................................185 

Williams, James A. .............................................. 186, 261, 263, 616 

Williams, Regina R. ...........................................................................613 

Williams, Richard D. .........................................................................374 

Wilson, D. U. ............................................................................................40 

Wilson, Heather .................................................................................400 

Wilson, James ...........................................................................108, 444 

Wilson, James M. ............................................................. 71, 128, 360 

Wilson, John R. ...................................................................................622 

Wilson, Matthew H. ..........................................................................269 

Wilson-Robles, Heather M. ...........................................................653 

Winslow, Monte .................................................................................436 

Wise, Megan ........................................................................................341 

Witt, William .......................................................................................281 

Witten, J .................................................................................................718 

Witting, Burghardt ...........................................................................686 

Wohlgenant, S. L. ...............................................................................381 

Wojno, Adam P. ...................................................................................231 

Wojton, Jeffrey ....................................................................................747 

Wold, William S. M. ...........................................................................480 

Wolf, Stephan ......................................................................................274 

Wolf, Walter .........................................................................................703 

Wolfe, John H. ............................................................................116, 289 

Wolfe, Thomas G. ...............................................................................550 

Wolfe, Tom ...........................................................................................339 

Wolstein, Orit .........................................................................................48 

Woltjen, Knut ......................................................................................715 

Won, Chong-Hyun .............................................................................602 

Wong, Jr, Kamehameha K. ..............................................................542 

Wong, Suet Ping .......................................................................346, 354 

Wong, Sunnie Y. ..................................................................................488 

Woo, Lee Hyun ...................................................................................699 

Wood, Steve .........................................................................................339 

Wood, Travis........................................................................................183 

Woodard, Lauren E. ..........................................................................269 


181


Woodle, Martin ...................................................................................455 

Wooley, Dawn P. .................................................................................267 

Worgall, Stefan ...................................................................................104 

Wourms, Michael ...............................................................................664 

Wright, J. Fraser ........................................ 110, 312, 529, 547, 663 

Wu, Bo ....................................................................................................451 

Wu, Chuanfeng ......................................................................................29 

Wu, Chu-Tse ...............................................................................105, 644 

Wu, Colin O. .............................................................................................65 

Wu, I.-Huan ..........................................................................................453 

Wu, James .............................................................................................659 

Wu, Jing .................................................................................................433 

Wu, Joseph ...........................................................................................474 

Wu, Lily ..................................................................................................215 

Wu, Ruey-Meei ......................................................................................60 

Wu, Ruilin .............................................................................................476 

Wu, Susan ................................................................................................87 

Wu, Wendy ...........................................................................................104 

Wu, Xiaolin ...........................................................................................308 

Wu, Xin ...................................................................................................749 

Wu, Zhijian ...........................................................................................709 

Würdinger, Thomas ..........................................................................321 

Wyszynski, Mike ................................................................................339 

Xia, Danny.............................................................................................184 

Xia, Xueqing .........................................................................................139 

Xiao, Bin ......................................................................................296, 367 

Xiao, Feng-jun ...........................................................................105, 644 

Xiao, Juan ..............................................................................................312 

Xiao, Ru ..................................................................................................330 

Xiao, Weidong .....................................................................................681 

Xiao, Xiao ..................................................... 167, 296, 367, 370, 449 

Xie, Jun .............................................................................................. 4, 512 

Xie, Qing ................................................................................................336 

Xie, Yi ......................................................................................................727 

Xie, Yili ...................................................................................................513 

Xie, Yuran ..............................................................................................711 

Xing, Elizabeth M. .................................................................................87 

Xu, Ding .................................................................................................448 

Xu, Lei .....................................................................................................296 

Xu, Weidong .........................................................................................555 

Xu, Wenqin ...........................................................................................524 

Xu, Yang ....................................................................................................49 

Xu, Zhili ..................................................................................................133 

Xue, Shu-Ya ...........................................................................................644 

Yadak, Rana ..........................................................................................521 

Yaguchi, Chiemi ..................................................................................228 

Yagyu, Shigeki .....................................................................................684 

Yakovief, Galina ..................................................................................495 

Yamada, Takeshi ................................................................................643 

Yamaguchi, Kiyoshi..............................................................................86 

Yamaguchi, Naoto .............................................................................216 

Yamamoto, Masato .................................................................408, 414 

Yamamoto, Masato .................................................................705, 742 

Yamamoto, Motoko ...........................................................................358 

Yamamoto, Shohei ...............................................................................86 

Yamamoto, Takashi ..........................................................................715 

Yamamoto, Yuki .................................................................................642 

Yamanaka, Shinya .............................................................................715 

Yamatmoto, Hirofumi ......................................................................749 

Yamazaki, Yoshiyuki .........................................................................601 

Yan, Jenny J. .............................................................................................46 

Yan, Jenny ...................................................................................183, 487 

Yan, Jian ............................................................................... 45, 341, 397 

Yan, Junjie .............................................................................................164 

Yan, Linna .............................................................................................750 

Yan, Pearlly...........................................................................................412 

Yan, Renhe ............................................................................................533 

Yan, Ziying ............................................................................................535 

Yancey, Heather ..................................................................................660 

Yáñez-Muñoz, Rafael J .....................................................................678 

Yang, Hsiao T. ......................................................................................166 

Yang, Hyunwoo ..................................................................................602 

Yang, Maria .................................................................................. 45, 341 

Yang, Qing .............................................................................................290 

Yang, Shan ............................................................................................216 

Yang, Shu ...............................................................................................374 

Yang, Xiaofang ....................................................................................512 

Yang, Yanqin ...........................................................................................65 

Yang, Yong ............................................................................................562 

Yang, Yue-feng ..........................................................................105, 644 

Yang, Yu-Lin .........................................................................................723 

Yannaki, Evangelia .................................................................... 27, 427 

182 



Yanovich, Saul .....................................................................................298 

Yaseen, Nahi Y. ....................................................................................647 

Yazicioglu, Mustafa ...........................................................................110 

Ye, Jing Jing ...........................................................................................476 

Ye, Jingjing ............................................................................................474 

Ye, Xun ....................................................................................................680 

Yeagy, Brian A. ....................................................................................493 

Yeger, Herman.....................................................................................433 

Yeghiazarians, Yerem .......................................................................369 

Yen, Paul M. ..........................................................................................618 

Yeoh, George C. T................................................................................356 

Yeung, Jonathan .................................................................................433 

Yezzi, Michael H. ................................................................................472 

Yin, Lu ......................................................................................................... 5 

Yin, Yue ..................................................................................................644 

Ying, Tang .............................................................................................614 

Ylä-Herttuala, Seppo ...........................................................................97 

Yliperttula, M ......................................................................................732 

Yockman, James W. ...........................................................................645 

Yoder, Mervin C. .......................................................................328, 542 

Yokoi, Kentaro ....................................................................................753 

Yokoi, Takayuki ..................................................................................753 

Yokoyama, Kazunari K. ...................................................................643 

Yoo, Hyuk Sang ...................................................................................256 

Yoo, James.............................................................................................253 

Yoo, Ji Young ........................................................................................747 

Yoon, Hye-Jin .......................................................................................602 

Yoon, Sea Young .................................................................................289 

Yoon, Sung Won .................................................................................143 

Yoshihara, Chieko ..............................................................................627 

Yoshioka, Akira ......................................................................................79 

You, Yezhi ..............................................................................................164 

Young, Brittany A. .............................................................................480 

Young, Courtney.................................................................................609 

Young, Sarah P. ....................................................................................618 

Youssouian, H. ......................................................................................15 

Yu, Feng .................................................................................................410 

Yu, Juehua ...................................................................................272, 659 

Yu, Jun-Ge .............................................................................................747 

Yu, Yang .......................................................................................206, 633 

Yu, Zhiqiang .........................................................................................164 

Yuan, Huijan ........................................................................................385 

Yuan, Zhenhua ............................................................... 367, 370, 449 

Yue, Yongping .............................................................................. 98, 613 

Yue, Yougping ......................................................................................166 

Yugeta, Naoko .....................................................................................608 

Yulyana, Yulyana ................................................................................254 

Yumul, Roma .............................................. 201, 303, 483, 552, 704 

Yun, Chae-Ok ................129, 130, 556, 634, 640, 645, 679, 689 

Yvon, Eric ...................................................................................... 12, 394 

Zabner, Joseph ....................................................................................327 

Zacchigna, Serena ..................................................................... 95, 368 

Zacharoulis, D .....................................................................................682 

Zaiss, Anne K. ......................................................................................448 

Zamorano-Carrillo, Absalom ........................................................ 604 

Zamore, Phillip D. ..............................................................................512 

Zanaboni, Laura .................................................................................420 

Zanatta, Daniela B. ............................................................................519 

Zanetti, Gianluigi .....................................................................309, 517 

Zaraspe, Kim .......................................................................................125 

Zaraspe, Kimberly ................................................................................39 

Zavaljevski, Maja ...............................................................................450 

Zeidan, Joumana ...................................................................................58 

Zelenaia, Olga ......................................................................................547 

Zentella-Dehesa, Alejandro ...........................................................534 

Zentilin, Lorena .....................................................................................95 

Zerfas, Patricia M. ..............................................................................383 

Zervou, Fani ............................................................................................27 

Zhang, Chao .........................................................................................562 

Zhang, Chunbo..........................................................................242, 750 

Zhang, Daoning .....................................................................................44 

Zhang, Fang ................................................................................313, 661 

Zhang, Feijie ........................................................................................271 

Zhang, Feng ..........................................................................................677 

Zhang, G. ...................................................................................................53 

Zhang, Jane ..............................................................................................48 

Zhang, Jianying ...................................................................................747 

Zhang, Jingang ..........................................................................533, 563 

Zhang, Jingwan ...................................................................................559 

Zhang, Jingyu.......................................................................................217 

Zhang, Jun .............................................................................................136 

Zhang, Keqing ................................................................... 98, 166, 613 


183


Zhang, Lei ..................................................................................... 46, 184 

Zhang, Ming ............................................................................................49 

Zhang, Mingdi ................................................................ 285, 478, 591 

Zhang, Na ..............................................................................................563 

Zhang, Qun-wei ........................................................................105, 644 

Zhang, Ting ..........................................................................................449 

Zhang, Tracy Y. .......................................................................................93 

Zhang, Wenli ........................................................................................557 

Zhang, Xiao-Bing ...............................................................................468 

Zhang, Xichen ......................................................................................239 

Zhang, Yan ............................................................................................369 

Zhang, Yanling ....................................................................................221 

Zhang, YiYi ...........................................................................................729 

Zhang, Yu ...............................................................................4, 128, 411 

Zhang, Yuanyuan ...............................................................................473 

Zhang, Zhao .........................................................................................247 

Zhang, Zhenwei ..................................................................................555 

Zhao, Allan ...........................................................................................281 

Zhao, Chunli .........................................................................................174 

Zhao, Chunxia ...........................................................................370, 449 

Zhao, Hui Fen ......................................................................................424 

Zhao, Huiren ..............................................................................339, 550 

Zhao, Li ..................................................................................................105 

Zhao, Na ......................................................................................207, 404 

Zhao, Yangbing ...................................................................................623 

Zhao, Yuan ............................................................................................526 

Zheng, Changyu ..................................................................................743 

Zheng, Dexian .....................................................................................752 

Zheng, Qifa ...........................................................................................214 

Zheng, X. Long ....................................................................................312 

Zheng, Yanyan .....................................................................................436 

Zheng, Zhaohui ...................................................................................298 

Zhong, Li ............................................................4, 128, 326, 328, 542 

Zhou, Betty ...........................................................................................455 

Zhou, Dongming ................................................................................562 

Zhou, Guisheng .........................................................................272, 659 

Zhou, Jiehua .........................................................................................454 

Zhou, Michelle ....................................................................................115 

Zhou, Qinghui......................................................................................164 

Zhou, Shangzhen ...................................... 110, 312, 488, 498, 733 

Zhou, Shang-Zhen .............................................................................547 

Zhou, Sheng .........................................................................................671 

Zhu, Liwei ...................................................................................207, 404 

Zimmermann, Wolfgang .................................................................658 

Zingone, Adriana ...............................................................................531 

Zolotukhin, Irene ...............................................................................190 

Zolotukhin, Sergei ...................................................................335, 548 

Zoltick, Philip K. .................................................................................523 

Zou, Jizhong ............................................................................................82 

Zourelias, Lee ............................................................................141, 149 

Zwaka, Thomas ..................................................................................169 

184

Final Program - American Society of Gene & Cell Therapy

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