Final Program - American Society of Gene & Cell Therapy

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Abstract Directory, Thursday, May 16, 2013 Poster Session I, continued 4:00 PM - 6:00 PM Room: Exhibit Hall C/D Late Breaking Abstracts I 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cells for TRAIL-induced apoptosis by downregulated XIAP Ahmet Cingoz, Yavuz Taga, Betul Catalgol 698 Abstract Withdrawn from Presentation 699 Culture and cryopreservation of human epidermal melanocytes (MC) for preclinical trials Kim Jin Tac, Pak Seoung Hoon, Shin Back Soo, Han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 Thursday, May 16, 2013 Final Program SALT LAKE CITY, UTAH May 15–18, 2013 107
Friday, May 17, 2013 Abstract Directory, Friday, May 17, 2013 Oral Abstract Session 320 DNA Vectorology & Gene Targeting 1:00 PM - 3:00 PM Room: Ballroom A 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. Oral Abstract Session 321 Gene & Cell Therapy of Diabetes, Metabolic and Genetic Diseases I 1:00 PM - 3:00 PM Room: Ballroom B 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 Therapy 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 Epidermolysis Bullosa 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 Final Program SALT LAKE CITY, UTAH May 15–18, 2013
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ASGCT Officers and Board of Directo
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Annual Meeting Exhibitors - as of A
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Annual Meeting Supporters Patron Le
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Committee Rosters Advisory Council
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Committee Rosters Host Committee Ch
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Committee Rosters Program Committee
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Abstract Reviewers Cancer - Immunot
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Outstanding New Investigator Awards
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Faculty List Thomas W. Chalberg, Jr
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Faculty List Ann M. Leen, PhD Baylo
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Faculty List Evan Y. Snyder, MD PhD
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General Meeting Information Continu
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General Meeting Information Speaker
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Salt Palace Convention Center Floor
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Salt Palace Convention Center Floor
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Program Schedule, Wednesday, May 15
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Program Schedule, Thursday, May 16,
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Page 80 and 81: Exhibitor Descriptions Aldevron 323
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Abstract Author Index Jacobs, Edwin
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Abstract Author Index Yanovich, Sau
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Final Program - American Society of Gene & Cell Therapy

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