FINAL PROGR AM - American Society of Gene & Cell Therapy

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AMERICAN SOCIETY OF GENE THERAPY • Final Program 53 ABSTRACTS THURSDAY, JUNE 2 AAV Vectors: Disease Applications 110 Performance of Different AAV Serotype Vectors Following Injection into the Deep Cerebellar Nuclei of ASMKO Mouse Brain James C. Dodge, Jennifer Clarke, Anthony Song, Jie Bu, Qi Zhao, Tatyana V. Taksir, Denise Griffiths, Lamya S. Shihabuddin, Catherine R. O’Riordan, Marco A. Passini Ed H. Schuchman, Gregory R. Stewart 111 AAV7, 8, and 9 Are More Efficient and Less Immunogenic Vectors for Muscle-Directed Gene Therapy for Hemophilia B Lili Wang, Jean-Pierre Louboutin, Yan Li, James M. Wilson 112 Strong Transgene-Specific Immune Responses Can Be Elicited with Novel Adeno-Associated Virus Vectors in Mice Yan Zhi, Julie Johnston, Di Wu, Joanita Figueredo, Martin Lock, Peter Bell, Guangping Gao, James M. Wilson 113 Manipulating AAV2 Tropism for Enhanced Delivery of AAV Vectors to the Spinal Cord Adam Davis, Matthew Stachler, Wenfang Shi, James Liu, Nicholas Boulis, Jeffrey Bartlett 114 Efficient Arterial Formation at the Sites of Adult Neo- Angiogenesis Requires the Recruitment of Bone Marrow Cells through the Neuropilin-1 (NP-1) Receptor Serena Zacchigna, Nikola Arsic, Lucia Pattarini, Silvia Moimas, Alessandro Carrer, Lorena Zentilin, Alessandro Salvi, Gianfranco Sinagra, Mauro Giacca 115 In Vivo Expression of Human Cob(I)alamin Adenosyltransferase Using rAAV Serotypes 1,2 and 8, an Approach to Gene Therapy for Methylmalonic Aciduria (MMA) Kirsten E. Erger, Thomas J. Conlon, Travis L. Cossette, Thomas A. Bobik, Terence R. Flotte 116 Functional Comparison of Four CFTR rAAV Vectors with Alternative Transcriptional Elements in Cell Lines and Polarized Human CF Airway Epithelia Liang Zhang, Benjamin Dutzar, Keith Munson, Barrie Carter, John F. Engelhardt 117 AAV-Mediated Erythropoietin Gene Transfer Protects from Genetic and Light-Induced Retinal Degeneration Mariacarmela Allocca, Tonia S. Rex, Luciano Domenici, Jean Bennet, Victor M. Rivera, Alberto Auricchio 118 Self-Complementary AAV Vectors Pseudotyped with Either Serotype 5 or 8 Capsids Give Therapeutic Human Factor IX Levels at Significantly Reduced Doses in Non- Human Primates John T. Gray, Andrew M. Davidoff, Amit C. Nathwani 119 Intraperitoneal Transduction of Adeno-Associated Virus 2 Expressing Angiostatin and Endostatin Synergistically Augments Paclitaxel Therapy and Tumor-Free Survival in a Mouse Model of Epithelial Ovarian Cancer Tatyana Isayeva, Changchun Ren, Selvarangan Ponnazhagan 120 Stimulation of the Synthesis of Type-II Collagen and Extracellular Matrix in Human Normal and Osteoarthritic Articular Cartilage Explants In Situ by Overexpression of Human sox9 by rAAV-Mediated Gene Transfer Henning Madry, Tanja Thurn, Dieter Kohn, Ernest F. Terwilliger, Magali Cucchiarini 121 VEGF-Induced Angiogenesis Does Not Have a Favorable Effect on Muscle Perfusion: A Relevant Role of Long- Term Angiopoietin-1 Expression in the Formation of Functional Blood Vessels Serena Zacchigna, Ennio Tasciotti, Nikola Arsic, Oreste Sorace, Marini Cecilia, Silvia Pardini, Debora Petroni, Claudia Kusmic, Lucia Pattarini, Silvia Moimas GianMario Sambuceti, Mauro Giacca 122 Enhanced Expression of Glutamate Decarboxylase 65 Significantly Improves Rat Parkinsonian Symptom Boyoung Lee, Young Ran Nam, Sung Jin Kim, Jin Hwan Oh, Yoon Hee Choi, Jin Woo Chang, Heuiran Lee 123 rAAV-pdx-1 Augmented the Insulin-Producing Cells Hua Li, Karen S. Lam, Sidney Tam, Weidong Xiao, Paul Tam, Ruian Xu 124 The “Perivascular Pump” Driven by Arterial Pulsation Is a Powerful Mechanism for the Distribution of Therapeutic Molecules within the Brain Piotr Hadaczek, Hanna Mirek, Laszlo Tamas, Martha C. Bohn, Charles Noble, John W. Park, Krystof Bankiewicz 125 AAV Vector-Mediated Neonatal Gene Transfer: Efficient Transgene Expression in Muscles after Intraperitoneal Cavity Vector Injection Tsuyoshi Ogura, Hiroaki Mizukami, Jun Mimuro, Takashi Okada, Hiromi Hamada, Akihiro Kume, Hiroyuki Yoshikawa, Yoichi Sakata, Keiya Ozawa
54 8th Annual Meeting • June 1 - 5, 2005 • St. Louis ABSTRACTS THURSDAY, JUNE 2 126 The Formation of Stable and Mature Blood Vessels Requires the Proper Timing of VEGF Expression Sabrina Tafuro, Serena Zacchigna, Lorena Zentilin, Marina Dapas, Mauro Giacca 127 Intraarticular Gene Transfer of Rat TNFR:Fc Suppressed CIA Arthritis Ai Zhi Zhao, Kai Gao, Xiao Yan Dong, Jian Qiang Peng, Xiao Bing Wu 128 Transduction of Hepatic Oval Cells, Stem Cells, In Vitro by Adeno-Associated Virus Vectors Hong You, Ping Wang, Jidong Jia, Liu Yong, Paul L. Hermonat 129 Development of Vectors Utilizing Strong Neuronal Cell Type Specific Promoters for High Level Expression of Beta Glucuronidase in the Central Nervous System Tupur Husain, John H. Wolfe 130 Characterization of a Murine Adeno-Associated Virus (AAV-mo.1) Capsid Michael Lochrie, Gwen Tatsuno, Alejandra Arbetman, Peter Smith, Jennifer Wellman, Shang-zhen Zhou, Glenn Pierce, Peter Colosi 131 Recombinant Adeno-Associated Virus 2-Mediated Transfer of the Human Superoxide-Dismutase Gene Does Not Confer Radioresistance on HeLa Cervical Carcinoma Cells Marlon R. Veldwijk, Carsten Herskind, Stephanie Laufs, W. Jens Zeller, Stefan Fruehauf, Frederik Wenz 132 An Optimized CsCl Gradient -Based Method to Generate High Concentration AAV Vector Stocks of High Purity and In Vivo Potency Mario Cooper, Bernd Hauck, Jeff Watkins, Megan Erne, Federico Mingozzi, Roland Herzog, Fraser Wright 133 Comparison of Adeno-Associated Viral Vector Serotypes 1, 2, 5 and 6 for Gene Transfer to Neurons and Glia In Vitro Doug B. Howard, Juan Li, Yun Wang, Brandon K. Harvey 134 Toxicology and Biodistribution Studies of a Recombinant Adeno-Associated Virus 1 (rAAV1) Alpha-1 Antitrypsin (AAT) Vector Amy E. Poirier, Thomas J. Conlon, Lynn A. Combee, Kirsten E. Erger, Terence R. Flotte 135 AAV-Mediated Gene Delivery To Evaluate the Biology of an Inherited Macular Degeneration Nicholas W. Keiser, Daniel C. Chung, Waixing Tang, Zhanyong Wei, Albert Maguire, Jean Bennett, Jeannette L. Bennicelli 136 Recombinant Adeno-Associated Virus 2-Mediated Ectopic Expression of að4bð1 Integrin in Mouse Mesenchymal Stem Cells Enhances Repopulation to Bone upon Syngeneic Transplantation in an Immunocompetent Mouse Model Sanjay Kumar, Selvarangan Ponnazhagan Adenovirus Vectors: General Biology and Host Response 137 Localization of Regions in CD46 That Interact with Adenovirus Anuj Gaggar, Dmitry M. Shayakhmetov, M. Kathryn Liszewski, John P. Atkinson, Andre Lieber 138 Blood Factors Affect Adenovirus Infectivity and Bio- Distribution In Vivo Dmitry M. Shayakhmetov 139 Priming and Boosting Efficiency of Chimp and Human Adenoviral Vectors in the Setting of Pre-Existing Immunity in Non-Human Primates Joanita Figueredo, Roberto Calcedo, Jim Miller, Gary Kobinger, Rebecca Grant, Guangping Gao, James M. Wilson 140 Primary Adenovirus Specific Cytotoxic T Lymphocyte Response Occurs after Viral Clearance and Liver Enzyme Elevation Jian Chen, Allan J. Zajac, Sylvia A. McPherson, Xin Xu, PingAr Yang, Qi Wu, David T. Curiel, John D. Mountz 141 Adenovirus Vaccine to Ebola Virus: Mechanism of Protection and Impact of Pre-Existing Immunity to the Vaccine Carrier Gary P. Kobinger, Heinz Feldmann, Yan Zhi, Gregory Schumer, Guangping Gao, Frederik Feldmann, Steven Jones, James M. Wilson 142 Molecular and Macromolecular Alterations of Recombinant Adenoviral Vectors Do Not Eliminate Changes in Hepatic Drug Metabolism Shellie M. Callahan, Michael P. Boquet, Piyanuch Wonganan, Maria A. Croyle
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156 NOTES 8th Annual Meeting • Ju
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FINAL PROGR AM - American Society of Gene & Cell Therapy

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