FINAL PROGRAM - American Society of Gene & Cell Therapy

13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 89Abstract DirectoryThursday, May 20, 2010Oral Abstract Session 235 — continuedImmunologic & Host Responses in Gene & Cell Therapy2:00 PM - 4:00 PMRoom: Virginia Suite44 2:45 PMLong-Term Phenotypic Correction of Hemophilia A after NeonatalAdministration of AAVChuhong Hu, Fides D. Lay, Joseph Springer, Ronald W. Busuttil, Gerald S. Lipshutz.45 3:00 PMImpact of Pre-Existing Neutralizing Antibodies on AAV8-Mediated,Liver-Directed Gene Transfer and Vector Biodistribution in NonhumanPrimatesLili Wang, Roberto Calcedo, Peter L. Bell, Martin W. Lock, Rebecca L. Grant, James M.Wilson.46 3:15 PMAutologous Dendritic Cell Therapy for Tolerance InductionGautam Sule, Masataka Suzuki, Racel Cela, John Rodgers, Brendan Lee.47 3:30 PMDifferential Transgene Expression in Dendritic Cells with UniqueRegulatory Cassettes Impacts Efficacy in Pompe DiseaseBaodong Sun, Ping Zhang, Xiao Yi Yang, Timothy C. Clay, Dwight D. Koeberl.48 3:45 PMImpact of the Host Environment on the Control of Gene Transfer-Induced Immune Responses with miRNA142.3p-Regulated VectorsFlorence Boisgérault, David-Alexandre Gross, Maxime Ferrand, Sylvie Darocha, JérômePoupiot, Marc Bartoli, Isabelle Richard, Anne Galy.Oral Abstract Session 236Oligonucleotide & RNAi Therapeutics I2:00 PM - 4:00 PMRoom: Delaware Suite49 2:00 PMSystemic Delivery of Antisense Morpholino Corrects RNA Mis-Splicing and Reduces Myotonia in a Transgenic Mouse Model ofMyotonic Dystrophy Type 1Thurman M. Wheeler, Yong-Fu Li, Krzysztof Sobczak, Paul A. Morcos, Charles A.Thornton.50 2:15 PMTriplex-Induced Correction of IDUA Gene Mutations as a Novel HurlerSyndrome TherapyJake del Campo, Karla D’Agostino, Ranjit S. Bindra, Peter M. Glazer.51 2:30 PMUsing rAAV Delivered miRNAs To Knockdown Misfolded HumanAlpha 1 Antitrypsin in a Transgenic Mouse ModelChristian Mueller, Qiushi Tang, Bryan O’Sullivan Murphy, Terence R. Flotte.52 2:45 PMConstitutive Long-Term Expression of shRNA in Liver Induces CellularStressMiwon Ahn, Scott R. Witting, Rafaela Ruiz, Romil Saxena, Nuria Morral.53 3:00 PMIn Vivo AAV Delivered Allele Specific shRNA for the Knockdown ofAlpha-1 AntitrypsinChristian Mueller, Bryan O’Sullivan-Murphy, Qiushi Tang, Terence R. Flotte.54 3:15 PMExpression Determinants of Mammalian Argonaute Proteins inMediating Gene SilencingPaul N. Valdmanis, Shuo Gu, Praveen Sethupathy, Mark A. Kay.55 3:30 PMAntisense Oligonucleotide-Mediated Therapy Increases SMN Levelsin the CNS and Prolongs Survival in a Severe Mouse Model of SpinalMuscular AtrophyMarco A. Passini, Jie Bu, Amy M. Richards, Lisa M. Stanek, S. P. Sardi, Yimin Hua, GeneHung, Lamya S. Shihabuddin, C. F. Bennett, Adrian R. Krainer, Seng H. Cheng.56 3:45 PMMultiple Exon-Skipping Using Cell-Penetrating Morpholinos forDystrophic DogsToshifumi Yokota, Takashi Saito, Nobuyuki Urasawa, Tetsuya Nagata, Akinori Nakamura,Ryszard Kole, Peter Sazani, Terence Partridge, Shin’ichi Takeda, Eric Hoffman.Poster Session I6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-SouthAAV Vectors I57Enveloped AAV Vectors for Gene Transfer ApplicationsCasey A. Maguire, Lucia Mincheva-Nilsson, Vladimir Baranov, Bakhos A. Tannous, MiguelSena-Esteves, Xandra O. Breakefield, Johan Skog.58Chimpanzee-Derived Novel Natural Variants of AAV9: VectorDevelopment and Interrogation of Correlations between CapsidStructure and Vector BiologyLi Zhong, Jun Xie, Qing Xie, Kim Van Vliet, Qiang Wang, Mengxin Li, Qin Su, Ran He,Jason Goetzmann, Mavis Mckenna, Terry Flotte, Guangping Gao.59Gene Delivery to the Pig Cone-Enriched Retina Highlights Species-Specific Differences in AAV-Mediated Photoreceptor TransductionClaudio Mussolino, Francesco Viola, Michele Della Corte, Settimio Rossi, Umberto DiVicino, Simona Neglia, Monica Doria, Francesco Testa, Roberto Giovannoni, ManuelaCrasta, Massimo Giunti, Edoardo Villani, Marialuisa Lavitrano, Maria Laura Bacci,Roberto Ratiglia, Francesca Simonelli, Alberto Auricchio, Enrico M. Surace.Thursday, May 20 th