13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 89Abstract DirectoryThursday, May 20, 2010Oral Abstract Session 235 — continuedImmunologic & Host Responses in <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>2:00 PM - 4:00 PMRoom: Virginia Suite44 2:45 PMLong-Term Phenotypic Correction <strong>of</strong> Hemophilia A after NeonatalAdministration <strong>of</strong> AAVChuhong Hu, Fides D. Lay, Joseph Springer, Ronald W. Busuttil, Gerald S. Lipshutz.45 3:00 PMImpact <strong>of</strong> Pre-Existing Neutralizing Antibodies on AAV8-Mediated,Liver-Directed <strong>Gene</strong> 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 <strong>Cell</strong> <strong>Therapy</strong> for Tolerance InductionGautam Sule, Masataka Suzuki, Racel Cela, John Rodgers, Brendan Lee.47 3:30 PMDifferential Transgene Expression in Dendritic <strong>Cell</strong>s with UniqueRegulatory Cassettes Impacts Efficacy in Pompe DiseaseBaodong Sun, Ping Zhang, Xiao Yi Yang, Timothy C. Clay, Dwight D. Koeberl.48 3:45 PMImpact <strong>of</strong> the Host Environment on the Control <strong>of</strong> <strong>Gene</strong> 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 <strong>of</strong> Antisense Morpholino Corrects RNA Mis-Splicing and Reduces Myotonia in a Transgenic Mouse Model <strong>of</strong>Myotonic Dystrophy Type 1Thurman M. Wheeler, Yong-Fu Li, Krzyszt<strong>of</strong> Sobczak, Paul A. Morcos, Charles A.Thornton.50 2:15 PMTriplex-Induced Correction <strong>of</strong> IDUA <strong>Gene</strong> Mutations as a Novel HurlerSyndrome <strong>Therapy</strong>Jake 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 <strong>of</strong> shRNA in Liver Induces <strong>Cell</strong>ularStressMiwon Ahn, Scott R. Witting, Rafaela Ruiz, Romil Saxena, Nuria Morral.53 3:00 PMIn Vivo AAV Delivered Allele Specific shRNA for the Knockdown <strong>of</strong>Alpha-1 AntitrypsinChristian Mueller, Bryan O’Sullivan-Murphy, Qiushi Tang, Terence R. Flotte.54 3:15 PMExpression Determinants <strong>of</strong> Mammalian Argonaute Proteins inMediating <strong>Gene</strong> SilencingPaul N. Valdmanis, Shuo Gu, Praveen Sethupathy, Mark A. Kay.55 3:30 PMAntisense Oligonucleotide-Mediated <strong>Therapy</strong> Increases SMN Levelsin the CNS and Prolongs Survival in a Severe Mouse Model <strong>of</strong> SpinalMuscular AtrophyMarco A. Passini, Jie Bu, Amy M. Richards, Lisa M. Stanek, S. P. Sardi, Yimin Hua, <strong>Gene</strong>Hung, Lamya S. Shihabuddin, C. F. Bennett, Adrian R. Krainer, Seng H. Cheng.56 3:45 PMMultiple Exon-Skipping Using <strong>Cell</strong>-Penetrating Morpholinos forDystrophic DogsToshifumi Yokota, Takashi Saito, Nobuyuki Urasawa, Tetsuya Nagata, Akinori Nakamura,Ryszard Kole, Peter Sazani, Terence Partridge, Shin’ichi Takeda, Eric H<strong>of</strong>fman.Poster Session I6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-SouthAAV Vectors I57Enveloped AAV Vectors for <strong>Gene</strong> Transfer ApplicationsCasey A. Maguire, Lucia Mincheva-Nilsson, Vladimir Baranov, Bakhos A. Tannous, MiguelSena-Esteves, Xandra O. Breakefield, Johan Skog.58Chimpanzee-Derived Novel Natural Variants <strong>of</strong> AAV9: VectorDevelopment and Interrogation <strong>of</strong> 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.59<strong>Gene</strong> 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
90<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryThursday, May 20 thThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-SouthAAV Vectors I60Enhancing AAV2 Transduction by Anti-Microtubule Drugs throughRhoA GTPase ActivationPing-Jie Xiao, Richard J. Samulski.61Tyrosine Mutations in the Capsid <strong>of</strong> AAV Vectors Alters TransductionProperties in the RetinaHilda Petrs-Silva, Astra Dinculescu, Seok H. Min, Li Zhong, Sergei Zolotukhin, ArunSrivastava, Alfred Lewin, William W. Hauswirth.62Riding Along with AAV: Mechanistic Studies on Vesicular TraffickingPathways <strong>of</strong> AAV SerotypesShen Shen, Xin Ming, Aravind Asokan, Rudy L. Juliano.63Effects <strong>of</strong> DNA Methylation on AAV Integration and TransgeneExpressionDiptiman Chanda, Jonathan A. Hensel, Jerome T. Higgs, Niroop Kaza, Rajat Grover,Selvarangan Ponnazhagan.64Efficient Transduction <strong>of</strong> Mouse Liver and Other Organs FollowingIntravenous Administration <strong>of</strong> a Recombinant Porcine AAVAlexander J. Bello, Allan R. Chand, Monica Doria, Kaylie Tran, Mariacarmela Allocca,Alberto Auricchio, Gary Kobinger.65Rational Design Meets Directed Evolution for Tailoring the AAVCapsidLuk H. Vandenberghe, Ru Xiao, Christie L. Bell, Kim Van Vliet, Jing Lu, Mavis Agbandje-McKenna, James M. Wilson.66AAV5 Transduction and Transgene Expression and Bioactivity inFibroblast-Like Synoviocytes from Rheumatoid Arthritis PatientsCaroline J. Aalbers, Scott A. Loiler, Federico Mingozzi, Paul-Peter Tak, Margriet J.Vervoordeldonk.67Pre-Injection <strong>of</strong> Polymers Can Increase the In Vivo TransductionEfficiency <strong>of</strong> Adeno-Associated Virus Type 2Gilles Moulay, Sylvie Boutin, Carole Masurier, Daniel Scherman, Antoine Kichler.68Therapeutic Expression <strong>of</strong> FIX at Reduced Doses Using AAV-ProteinPhosphotase-5 Helper VirusBrandon K. Sack, Jayandharan R. Giridhara, Zhong Li, Herzog W. Roland, SrivastavaArun.69A Scalable Process for Purification <strong>of</strong> Recombinant Adeno-AssociatedVirus Serotype 9Jingmin Zhou, Xiao Yang, Linda Couto, Katherine A. High, Guang Qu.70Adeno- Associated Virus Type 2 Rep Proteins Mediate PreferentialIntegration <strong>of</strong> a Lentiviral VectorVictor J. McAlister, Tshaka Cunningham, Anthony T. Craig, Roland A. Owens.71High Throughput Assay for Quantifying In Vitro AAV VectorTransduction Efficiency in ARPE19 <strong>Cell</strong>sRenee C. Ryals, Sanford L. Boye, Shannon Boye, Andrew Neely, William W. Hauswirth.72Optimization <strong>of</strong> Parameters for AAV Production in Insect <strong>Cell</strong>s andConditions for Baculovirus StorageHaifeng Chen.Adenovirus and Other DNA Virus Vectors I73Radiation-Induced Liver Fibrosis Is Mitigated by <strong>Gene</strong> <strong>Therapy</strong>Against Transforming Growth Factor-b Signaling in the RatDu Shi-Suo, Zeng Zhao-Chong, Zhou Jian, Tan Yun-shan, Zhang Zheng-Yu, Zeng Hai-Ying,Liu Zhong-shan.74Protection <strong>of</strong> Salivary Glands from Irradiation Damage by hMnSOD<strong>Gene</strong> TransferAna P. Cotrim, Anne M. Rowzee, A. Sowers, James B. Mitchell, Bruce J. Baum.75Intrathecal Injection <strong>of</strong> HDAd Vectors Results in Long-Term TransgeneExpression in Neuroepithelial <strong>Cell</strong>s and NeuronsPasquale Piccolo, Scott Dindot, Nathan Grove, Donna Palmer, Nicola Brunetti-Pierri.76Virion-Associated Co-Factor HMGB1 Facilitates Transposition fromthe HSV/Sleeping Beauty Amplicon Vector PlatformSuresh de Silva, Louis T. Lotta, Clark A. Burris, William J. Bowers.77hKGF <strong>Gene</strong> Transfer for the Prevention <strong>of</strong> Radiation-Induced SalivaryHyp<strong>of</strong>unctionChangyu Zheng, Ana P. Cotrim, Anne M. Rowzee, William D. Swaim, Anastasia Sowers,James B. Mitchell, Bruce J. Baum.78<strong>Gene</strong>ration <strong>of</strong> Replication-Defective Adenovirus Vector ExpressingHCV Structural <strong>Gene</strong> CE1E2 and Comparative Immunogenicity in Micewith Different Delivery RoutesJie Guan, Yao Deng, Xiao Yin, Wenjie Tan, Xiaobing Wu.EXHIBITOR PROSPECTUSfinal program
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