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FINAL PROGR AM - American Society of Gene & Cell Therapy

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<strong>AM</strong>ERICAN SOCIETY OF GENE THERAPY • Final Program 95<br />

ABSTRACTS<br />

SATURDAY, JUNE 4<br />

808 11:30 <strong>AM</strong><br />

Monitoring <strong>of</strong> Transgene Expression in Cancer Patients<br />

with Positron Emission Tomography (PET)<br />

Bruno Sangro, Ivan Penuelas, Guillermo Mazzolini, Jose F.<br />

Boan, Josep M. Marti-Climent, Maria Ruiz, Cheng Qian,<br />

Nagichettiar Satyamurthy, Jorge R. Barrio, Michael E. Phelps<br />

Jose A. Richter, Sam Gambhir, Jesus Prieto<br />

809 11:45 <strong>AM</strong><br />

mda-7 Kills Pancreatic Cancer <strong>Cell</strong>s by Inhibition <strong>of</strong> Wnt/<br />

PI3K Signaling: Identification <strong>of</strong> IL-20 Receptor-<br />

Mediated Bystander Activity Against Pancreatic Cancer<br />

Sunil Chada, Dora Bocangel, Ramesh Rajagopal, Elizabeth<br />

Grimm, Abner M. Mhashilkar, Mingzhong Zheng<br />

810 12:00 PM<br />

Ex-Vivo Activation and <strong>Gene</strong>tic Manipulation <strong>of</strong> Human<br />

T <strong>Cell</strong>s Results in Consistent In-Vivo Expansion and<br />

Lethal GvHD in a Novel Murine Xenotransplant Model<br />

Bruno Nervi, Michael P. Rettig, Julie K. Ritchey, Gerhard<br />

Bauer, Jon Walker, Phillip E. Herrbrich, Todd E. Meyerrose,<br />

Mark Bonyhadi, Jan A. Nolta, John F. DiPersio<br />

Oral Abstract Session 417<br />

<strong>Gene</strong> <strong>Therapy</strong> Approaches to Pulmonary Disease<br />

Room: 261/262<br />

811 10:15 <strong>AM</strong><br />

ACE-Targeted eNOS and BMPR2 <strong>Gene</strong> <strong>Therapy</strong><br />

Attenuates Pulmonary Hypertension in a Chronic<br />

Hypoxia Rat Model<br />

Ann M. Reynolds, Mark D. Holmes, David T. Curiel, Andrew<br />

H. Baker, Paul N. Reynolds<br />

812 10:30 <strong>AM</strong><br />

Non-Human Primate Serotype rh.10 AAV Directed<br />

Therapeutic Serum Levels <strong>of</strong> að1-antitrypsin Following<br />

Intrapleural Administration in Mice with Pre-Existing<br />

Immunity to AAV<br />

Bishnu P. De, Adriana Heguy, Neil R. Hackett, John Lee,<br />

Guangping Gao, James M. Wilson, Ronald G. Crystal<br />

813 10:45 <strong>AM</strong><br />

Distribution <strong>of</strong> Transgene Expressing <strong>Cell</strong>s Following<br />

Intramarrow Injection to Transduce Hematopoietic Stem<br />

<strong>Cell</strong>s In Situ Using rSV40 Vectors<br />

Jean-Pierre Louboutin, Bianling Liu, David S. Strayer<br />

814 11:00 <strong>AM</strong><br />

Adenovirus-Mediated <strong>Gene</strong> Transfer Demonstrates That<br />

Pirin, a Transcription Factor Up-Regulated in the<br />

Bronchial Epithelium by Cigarette Smoke, Mediates<br />

Bronchial Epithelial <strong>Cell</strong> Apoptosis<br />

Brian D. Gelbman, Adriana Heguy, Timothy P. O’Connor,<br />

Joseph Zabner, Ronald G. Crystal<br />

815 11:15 <strong>AM</strong><br />

Aerosol Delivery <strong>of</strong> Helper-Dependent Adenoviral<br />

Vector into Nonhuman Primate Lungs Results in High<br />

Efficiency Pulmonary Transduction with Minimal<br />

Toxicity<br />

Peter Hiatt, Nicola Brunetti-Pierri, David Koehler, Ruth<br />

McConnell, Julie Katkin, Donna Palmer, David Dimmock, Jim<br />

Hu, Milton Finegold, Arthur Beaudet Dee Carey, Karen Rice,<br />

Philip Ng<br />

816 11:30 <strong>AM</strong><br />

RNAi Applications for Respiratory Epithelia: Delivery<br />

and Efficacy<br />

Anthony J. Fischer, Christine L. Wohlford-Lenane, Patrick L.<br />

Sinn, Hong Peng Jia, Steven M. Varga, Beverly L. Davidson,<br />

Paul B. McCray<br />

817 11:45 <strong>AM</strong><br />

Protein Transduction Domain Mediated DFRD Correction<br />

<strong>of</strong> CFTR Function in Airway <strong>Cell</strong>s from DF508<br />

Homozygous CF Patients<br />

Zhibao Mi, Fei Sun, Xiaoli Lu, Steve Condliffe, Xiaoyan<br />

Gong, Joseph Pilewski, Raymond A. Frizzell, Paul D. Robbins<br />

818 12:00 PM<br />

Recombinant AAV1-Mediated Treatment <strong>of</strong> CNS<br />

Glycogen Accumulation in Mouse Model <strong>of</strong> Glycogen<br />

Storage Disease Type II<br />

Lara R. DeRuisseau, Cathryn S. Mah, Thomas J. Fraites, Jr.,<br />

David D. Fuller, Barry J. Byrne<br />

Presidential Symposium 430<br />

Room: America’s Ballroom<br />

819 3:20 PM<br />

Molecular Follow-Up <strong>of</strong> Patients Treated with Allogeneic<br />

Hematopoietic Stem <strong>Cell</strong> Transplantation and Donor<br />

Lymphocytes Transduced with a Retroviral Vector<br />

Expressing HSV-TK and ∆LNGFR<br />

Alessandra Recchia, Zulma Magnani, Fabrizia Urbinati,<br />

Daniela Sartori, Sara Muraro, Maria T. Lupo Stanghellini,<br />

Massimo Bernardi, Alessandra Pescarollo, Marco Bregni, Chiara<br />

Bonini Claudio Bordignon, Fabio Ciceri, Fulvio Mavilio<br />

820 3:38 PM<br />

Highly Efficient Exon-Skipping and Sustained Correction<br />

<strong>of</strong> Muscular Dystrophy Using an Adeno-Associated<br />

Viral Vector<br />

Aurelie Goyenvalle, Adeline Vulin, Jean Claude Kaplan, France<br />

Leturcq, Olivier Danos, Luis Garcia<br />

821 3:56 PM<br />

Development <strong>of</strong> a Site-Specific Integrating <strong>Gene</strong>-Deleted<br />

Adenoviral Vector for Sustained Transgene Expression<br />

Levels In Vivo<br />

Anja Ehrhardt, Stephen R. Yant, Giering A. Jeff, Hui Xu,<br />

Michele P. Calos, Mark A. Kay

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