Final Program - American Society of Gene & Cell Therapy

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Abstract Directory, Thursday, May 16, 2013 Oral Abstract Session 231, continued RNA Virus Vectors 2:00 PM - 4:00 PM Room: Ballroom B 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. Oral Abstract Session 232 AAV Vector Biology 2:00 PM - 4:00 PM Room: Ballroom C 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. Oral Abstract Session 233 Stem Cell Engineering and Therapy 2:00 PM - 4:00 PM Room: Ballroom D 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. Thursday, May 16, 2013 Final Program SALT LAKE CITY, UTAH May 15–18, 2013 93
Abstract Directory, Thursday, May 16, 2013 Oral Abstract Session 233, continued Stem Cell Engineering and Therapy 2:00 PM - 4:00 PM Room: Ballroom D Oral Abstract Session 234 Musculo-skeletal Gene & Cell Therapy I 2:00 PM - 4:00 PM Room: 150 DEFG Thursday, May 16, 2013 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 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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Abstract Author Index Bridges, Char
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Abstract Author Index Chicoine, Lou
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Abstract Author Index Daukandt, Mar
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Abstract Author Index Eleftheriadou
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Abstract Author Index Furth, Mark E
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Abstract Author Index Grosso, Chant
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Abstract Author Index Hickey, Ray D
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Abstract Author Index Jacobs, Edwin
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Abstract Author Index Ugai, Hideyo.
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Final Program - American Society of Gene & Cell Therapy

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