FINAL PROGR AM - American Society of Gene & Cell Therapy

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AMERICAN SOCIETY OF GENE THERAPY • Final Program 71 ABSTRACTS FRIDAY, JUNE 3 418 2:30 PM rAAV-Mediated Expression of Adiponectin Receptor (AdipoR2) cDNA in Muscle Prevents the Development of Obesity in DIO Rats George Aslanidi, Stanislav Shklayev, Erik Kohlbrenner, Vadim Kroutov, Glenn Walter, Martha Campbell-Thompson, Sergei Zolotukhin 419 2:45 PM Generation of Transgene Product-Specific Regulatory CD4+CD25+ T Cells by Hepatic AAV Gene Transfer O. Cao, E. Dobrzynski, B. L. Mingle, L. Wang, R. W. Herzog 420 3:00 PM Tropism-Modified Adeno-Associated Virus Vector Mediates Targeting of Brain Vascular Endothelium In Vivo Yong Hong Chen, Beverly L. Davidson 421 3:15 PM Liver Contains Sufficient Sulfatase Modifying Factor for 4-Sulfatase and Correction of MPS VI with Neonatal Retroviral Gene Therapy in Cats Mark Haskins, Thomas O’Malley, Bin Wang, Jason Metgcalf, Ping Wang, John Hopwood, Katherine P. Ponder 422 3:30 PM Dominant-Negative Interference in the Pah enu2 Mouse Model of PKU: Effectiveness of Vectors Expressing Either Modified Forms of Phenylalanine Hydroxylase (PAH) or Ribozymes Plus a Hardened PAH mRNA Catherine E. Charron, O. P. Perera, Stacy L. Porvasnik, Mandy Blackburn, Alfred S. Lewin, Philip J. Laipis 423 3:45 PM Increased Incidence of Hepatocellular Change and Neoplastic Disease in AAV-WPRE Treated Pah enu2 Mice Jennifer E. Embury, Catherine E. Charron, Philip J. Laipis 424 4:00 PM Intracranial AAV5 Synergizes with Non-Myeloablative Bone Marrow Transplantation in the Murine Model of Globoid-Cell Leukodystrophy Darshong Lin, Anthony Donsante, Shannon Macauley, Beth Levy, Carole Vogler, Mark Sands 425 4:15 PM AAV2-Mediated Expression of Iduronate Sulfatase in Peripheral Tissues and the CNS in Mucopolysaccharidosis II Mouse Model by Combined Intravenous and Intracisternal Vector Delivery Lu Kang, Haiyan Fu, Jerillyn S. Jennings, Joseph Muenzer Oral Abstract Session 334 Neurologic Disorders Room: 265/266 426 2:00 PM Cure of Established Disease and Direct Evidence of Cellular Cross-Correction in the Nervous System of Metachromatic Leukodystrophy Mice after HSC-Based Gene Therapy Alessandra Biffi, Alessia Capotondo, Angelo Quattrini, Ubaldo Del Carro, Riccardo Brambilla, Stefania Fasano, Sergio Marchesini, Claudio Bordignon, Luigi Naldini 427 2:15 PM AAV2- and AAV5-Mediated CNS Delivery of Human CLN2 Reduces Lysosomal Storage in a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis Marco A. Passini, Dolan Sondhi, James C. Dodge, Jie Bu, Wendy Yang, Neil R. Hackett, Stephen M. Kaminsky, Mukarram El-Banna, Qinwen Mao, Beverly L. Davidson David E. Sleat, Peter Lobel, Ronald G. Crystal, Gregory R. Stewart 428 2:30 PM A Novel Approach for the Treatment of the Neurological Symptoms of MPS VII (Sly Disease) Brian J. Spencer, Inder M. Verma 429 2:45 PM AAV5-Mediated Delivery of Human Aryl Sulfatase A (hARSA) Prevents Sufatide Storage and Neuropathological Phenotype in Metachromatic Leukodystrophy (MLD) Mice Nathalie Cartier, Caroline Sevin, Abdellatif Benraiss, Peter DeDeyn, Delphine Bonnin, Marie-T Vanier, Moullier Philippe, Volkmar Gieselmann, Patrick Aubourg 430 3:00 PM Immune System Regulation of Transgene Expression from High-Capacity Gutless Adenoviral Vectors in the Mouse Brain: Very Long Term Expression Following Pre- Immunization Carlos Barcia, Jeffrey M. Zirger, Weidong Xiong, Philip Ng, Donna Palmer, Maria G. Castro, Pedro R. Lowenstein 431 3:15 PM In Utero CNS Delivery of an Integrating HSV-1 Amplicon Vector Leads to Prolonged, Neuron-Specific Gene Expression William J. Bowers, Michael A. Mastrangelo, Ann E. Casey, Hilary A. Southerland, Kathleen A. Maguire-Zeiss, Howard J. Federoff 432 3:30 PM Molecular Chimerism Prevents Experimental Autoimmune Encephalomyelitis (EAE) Herena Eixarch, Carmen Espejo, Francisco Vidal, Silvia Garces, Mireia Castillo, Alex Bote, Elisabeth Kadar, Marta Rosal, Xavier Montalban, Jordi Barquinero
72 8th Annual Meeting • June 1 - 5, 2005 • St. Louis ABSTRACTS FRIDAY, JUNE 3 433 3:45 PM Gene Therapy of Epilepsy by Adenovirus-Mediated Tetanus Toxin Light Chain Gene Transfer Jun Yang, Qingshan Teng, Mary E. Garrity-Moses, Thais Federici, Imad Najm, Stephan Chabardes, Michael Moffitt, Nicholas M. Boulis 434 4:00 PM Long-Term Effects on Retinal Function and Structure Using AAV-Mediated Gene Therapy in Blind RPE65 Null Mutation Dogs Kristina Narfstrom, Greg Tullis, Ragnheidur Bragadottir, Mathias Seeliger, Elizabeth P. Rakoczy, Martin L. Katz 435 4:15 PM Functional and Morphological Rescue of the Type I Ocular Albinism Murine Retina Following AAV-Mediated Gene Transfer. Enrico M. Surace, Luciano Domenici, Katia Cortese, Consuelo Venturi, Gabriella Cotugno, Umberto Di Vicino, Alessandro Cellerino, Valeria Marigo, Carlo Tacchetti, Andrea Ballabio Alberto Auricchio Oral Abstract Session 335 Infectious Diseases and Vaccines: RNAi in Gene Therapy for Infections Room: 230/231 436 2:00 PM shRNAs Target HIV-1 Vif Gene Coding for SOCS-Box Motif Prevent Virus Escape from RNA Interference- Mediated Inhibition Jianfei Ji, John J. Rossi 437 2:15 PM Alternative Strategies To Deliver Multiple shRNAs in Order to Simultaneously Inhibit Multiple Targets Peter W. Roelvink, Michael W. Graham, David A. Suhy, Linda B. Couto, Sara M. Cunningham, Mark A. Kay, John J. Rossi, Alexander A. Kolykhalov 438 2:30 PM Development of an siRNA Based Therapy for Hepatitis Virus Infection David V. Morrissey, Karin Blanchard, Lucinda Shaw, Kristi Jensen, Wendy Breen, Shawn Zinnen, Brent Dickinson, James A. McSwiggen, Chandra Vargeese, Keith Bowman Chris S. Shaffer, Lloyd Jeffs, Adam Judge, Barry Polisky, Jennifer A. Lockridge, Amy C. H. Lee, Ian MacLachlan 439 2:45 PM CXCR4 and CCR5 shRNA Transgenic Macrophages Are Functionally Normal and Resist HIV-1 Infection Joseph Anderson, Ramesh Akkina 440 3:00 PM A Non-Human Primate Model for Lentivirus-Mediated Anti-HIV RNAi Strategies Karen Beagles, Grant Trobridge, Brian Beard, John Rossi, Jiing- Kuan Yee, Shiu-Lok Hu, Hans-Peter Kiem 441 3:15 PM Short Hairpin RNA Interference Expressed from a Gene- Deleted Adenoviral Vector Results in Reduction of Hepatits B Surface Antigen Levels In Vitro and in a Small Animal Model for Hepatitis B Infection Anja Ehrhardt, Hui Xu, Felix H. Salazar, Patricia L. Marion, Mark A. Kay 442 3:30 PM Evaluation of siRNA for Induction of In Vitro Resistance in HIV-1 Priscilla Yam, Jerry Wu, Ying Yu, John A. Zaia, Jiing-Kuan Yee 443 3:45 PM Tissue Specific Expression of Short Hairpin RNAs for the Treatment of HCV Using Liver Specific RNA Pol II Expression Cassettes David A. Suhy, Alexander A. Kolykhalov, Linda B. Couto, Luz Maria Garcia, A. Rosanna Schroeder, Amy E. Parker, Gabriel Haniff, Sara M. Cunningham, Mark A. Kay, Peter W. Roelvink Oral Abstract Session 336 Targeting of Adeno Viral Vectors and Cancer Treatment Room: 123/127 444 2:00 PM A New Vector Which Targets Breast Cancer and Its Vasculature Yanzheng Liu, Tao Ye, Jonathan Maynard, Hakan Akbulut, Albert Deisseroth 445 2:15 PM Combinatorial Antiangiogenic Gene Therapy by Nonviral Gene Transfer Using the Sleeping Beauty Transposon Causes Tumor Regression and Improves Survival in Mice Bearing Intracranial Human Glioblastoma John R. Ohlfest, Zachary L. Demorest, Yasuhiko Motooka, Isabelita Vengco, Seunguk Oh, Eleanor Chen, Frank A. Scappaticci, Stephen C. Ekker, Walter C. Low, Andrew B. Freese David A. Largaespada 446 2:30 PM Tie2 Expression Defines an Integrated System of Cell Types Specifically Involved in Angiogenesis, and Provides a Platform for Targeted Gene Delivery to Tumors Michele De Palma, Mary Anna Venneri, Rossella Galli, Nathalie Belmonte, Maurilio Sampaolesi, Lucia Sergi Sergi, Luigi Naldini
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156 NOTES 8th Annual Meeting • Ju
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