FINAL PROGRAM - American Society of Gene & Cell Therapy

13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010105Abstract DirectoryFriday, May 21, 2010Oral Abstract Session 320 I — continuedClinical Gene & Cell Therapy Oral Abstract Session2:00 PM - 4:00 PMRoom: Marriott Ballroom Salons 2 & 3296 3:45 PMUpdate on a Clinical Gene Therapy Trial for Adenosine DeaminaseDeficient Severe Combined Immune Deficiency (ADA-SCID)Kit L. Shaw, Yeong “Christopher” Choi, Linda Muul, Robert Sokolic, Denise A. Carbonaro,Alan Ikeda, Monika Smogorzewska, Jayashree Jagadeesh, Elizabeth Garabedian, AmiShah, Neena Kapoor, Michael S. Hershfield, Fabio Candotti, Donald B. Kohn.Oral Abstract Session 321RNA Virus Vectors2:00 PM - 4:00 PMRoom: Virginia Suite297 2:00 PMSensitive In Vivo Models To Assess the Risk of InsertionalMutagenesis in the Liver upon Vector Systemic DeliveryMarco Ranzani, Daniela Cesana, Manfred Schmidt, Francesca Sanvito, FabrizioBenedicenti, Cynthia Bartholomä, Maurilio Ponzoni, Alessio Cantore, Lucia Sergi Sergi,Claudio Doglioni, Christof VonKalle, Luigi Naldini, Eugenio Montini.298 2:15 PMSystemic Injection in a Tumor Prone Mouse Unravels the ResidualGenotoxicity of Self-Inactivating Lentiviral VectorsDaniela Cesana, Marco Ranzani, Cynthia Bartholomä, Manfred Schmidt, FabrizioBenedicenti, Monica Volpin, Christof VonKalle, Luigi Naldini, Eugenio Montini.299 2:30 PMSpecific Insertions of Zinc Finger Domains into Gag-Pol YieldsEngineered Retroviral Vectors with Selective Integration PropertiesKwang-il Lim, Ryan Klimczak, Julie Yu, David Schaffer.300 2:45 PMThe D4Z4 Insulator Protects Retrovirus Vectors from Silencing inStem CellsSylvie Rival-Gervier, Susan Anderson, Mandy Lo, Peter Pasceri, James Ellis.301 3:00 PMExpression of the LEDGF Integrase Binding Domain (IBD) SeverelyRestricts HIV-1 Replication and Drives Evolution of PhenotypicallyDistinctive Integrase MutantsDyana T. Saenz, Anne Meehan, Mary E. Peretz, Eric M. Poeschla.302 3:15 PMTransient and Highly Efficient Delivery of Sleeping BeautyTransposase and Flp Recombinase mRNA in Target Cells Via RT-Deficient Retroviral ParticlesMelanie Galla, Axel Schambach, Tobias Maetzig, Niels Heinz, Zsuzsanna Izsvak, ZoltanIvics, Christopher Baum.303 3:30 PMErasable Non-Integrating RNA Replicon Vectors for Generation ofImmaculate iPS Cells Free from Transgenes and Sequence-InterferenceHiroshi Ban, Noemi Fusaki, Toshiaki Tabata, Koichi Saeki, Akihiro Iida, Makoto Inoue,Mamoru Hasegawa.304 3:45 PMSelf-Inactivating Alpharetroviral Vectors with a Split-PackagingDesignJulia D. Sürth, Tobias Mätzig, Melanie Galla, Ute Modlich, Christopher Baum, AxelSchambach.Oral Abstract Session 322AAV Vectors: Vector Biology2:00 PM - 4:00 PMRoom: Marriott Ballroom Salon 1305 2:00 PMDissecting rAAV Integration Frequency in Non-Human Primates byDeep SequencingAli Nowrouzi, Magalie Penaud-Budloo, Christine Kaeppel, Anne Arens, Caroline Le Guiner,Adrien Leger, Karim Zaoui, Christian Weber, Richard O. Snyder, Christof von Kalle,Philippe Moullier, Manfred Schmidt.306 2:15 PMNon-Canonical Integrating AAV Vectors as a Safe Tool for the Futureof Gene TherapyLeszek Lisowski, Zhongya Wang, Ashley Lau, Feijie Zhang, Markus Grompe, Mark A.Kay.307 2:30 PMInteraction with Homologous Recombination Pathways Leads To Lossof Functional Recombinant AAV (rAAV) GenomesMarcela P. Cataldi, Douglas M. McCarty.308 2:45 PMHigh-Throughput Sequencing of AAV Vector Integrants To AssessPotential for GenotoxicityHojun Li, Nirav Malani, Shari R. Hamilton, Alexander Schlachterman, Giulio Bussadori,Shyrie E. Edmonson, Rachel Shah, Federico M. Mingozzi, J. F. Wright, Valder R. Arruda,Frederic D. Bushman, Katherine A. High.309 3:00 PMA Self-Complimentary rAAV Vector Genome but Not Variations in theCapsid Sequence Magnifies the Innate Immune Response FollowingLiver Directed DeliveryAshley T. Martino, Masataka Suzuki, Brendan Lee, Roland W. Herzog.310 3:15 PMA Proteomic Analysis of the Protein Composition in Recombinant AAVVector PreparationsBiao Dong, Xunbao Duan, Fayaz Khazi, Kirk Chu, Jihong Dai, Jenni A. Snyder, Hui Lu,Weidong Xiao.311 3:30 PMDownregulation of DNA-Damage Response Proteins in PostmitoticTissues Correlates with Increased AAV TransductionJasmina Lovric, Miguel Mano, Lorena Zentilin, Mauro Giacca.Friday, May 21 st