13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010105Abstract DirectoryFriday, May 21, 2010Oral Abstract Session 320 I — continuedClinical <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> Oral Abstract Session2:00 PM - 4:00 PMRoom: Marriott Ballroom Salons 2 & 3296 3:45 PMUpdate on a Clinical <strong>Gene</strong> <strong>Therapy</strong> 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 <strong>of</strong> 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, Christ<strong>of</strong> VonKalle, Luigi Naldini, Eugenio Montini.298 2:15 PMSystemic Injection in a Tumor Prone Mouse Unravels the ResidualGenotoxicity <strong>of</strong> Self-Inactivating Lentiviral VectorsDaniela Cesana, Marco Ranzani, Cynthia Bartholomä, Manfred Schmidt, FabrizioBenedicenti, Monica Volpin, Christ<strong>of</strong> VonKalle, Luigi Naldini, Eugenio Montini.299 2:30 PMSpecific Insertions <strong>of</strong> 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 <strong>Cell</strong>sSylvie Rival-Gervier, Susan Anderson, Mandy Lo, Peter Pasceri, James Ellis.301 3:00 PMExpression <strong>of</strong> the LEDGF Integrase Binding Domain (IBD) SeverelyRestricts HIV-1 Replication and Drives Evolution <strong>of</strong> PhenotypicallyDistinctive Integrase MutantsDyana T. Saenz, Anne Meehan, Mary E. Peretz, Eric M. Poeschla.302 3:15 PMTransient and Highly Efficient Delivery <strong>of</strong> Sleeping BeautyTransposase and Flp Recombinase mRNA in Target <strong>Cell</strong>s 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 <strong>Gene</strong>ration <strong>of</strong>Immaculate iPS <strong>Cell</strong>s 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, Christ<strong>of</strong> von Kalle,Philippe Moullier, Manfred Schmidt.306 2:15 PMNon-Canonical Integrating AAV Vectors as a Safe Tool for the Future<strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong>Leszek Lisowski, Zhongya Wang, Ashley Lau, Feijie Zhang, Markus Grompe, Mark A.Kay.307 2:30 PMInteraction with Homologous Recombination Pathways Leads To Loss<strong>of</strong> Functional Recombinant AAV (rAAV) GenomesMarcela P. Cataldi, Douglas M. McCarty.308 2:45 PMHigh-Throughput Sequencing <strong>of</strong> 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 <strong>of</strong> 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 <strong>of</strong> DNA-Damage Response Proteins in PostmitoticTissues Correlates with Increased AAV TransductionJasmina Lovric, Miguel Mano, Lorena Zentilin, Mauro Giacca.Friday, May 21 st
106<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryFriday, May 21 stFriday, May 21, 2010Oral Abstract Session 322 — continuedAAV Vectors: Vector Biology2:00 PM - 4:00 PMRoom: Marriott Ballroom Salon 1312 3:45 PMInnate Immune Response towards Adeno-Associated Viral (AAV)Vectors in Human Liver Non-Parenchymal <strong>Cell</strong>sMarianna Hoesel, Mathias Broxtermann, Hanna Janicki, Knud Esser, Silke Arzberger,Miriam John von Freyend, Dirk Stabenow, Percy Knolle, Michael Hallek, Ulrike Protzer,Hildegard Buening.Oral Abstract Session 323<strong>Gene</strong> <strong>Therapy</strong> for Lysosomal Storage Diseases andOther <strong>Gene</strong>tic Disorders2:00 PM - 4:00 PMRoom: Delaware Suite313 2:00 PMTowards a Safe and Effective <strong>Gene</strong> <strong>Therapy</strong> Approach for Globoid <strong>Cell</strong>LeukodystrophyIlaria Visigalli, Silvia Ungari, Bernhard Gentner, Sabata Martino, Daniela Ungaro, LetterioS. Politi, Lucia Sergi Sergi, Ubaldo Del Carro, Angelo Quattrini, Luigi Naldini, AlessandraBiffi.314 2:15 PMMesenchymal Stem <strong>Cell</strong> <strong>Therapy</strong> for the Twitcher Mouse Model <strong>of</strong>Krabbe’s DiseaseCynthia Ripoll, Mette Flaat, Jessica Eiermann, Jeanne Perkins, Brittni Scruggs, MarjorieMcCants, Michelle Eagle, William Wimley, Cynthia Trygg, Jeffrey Gimble, Bruce Bunnell.315 2:30 PMTherapeutic Efficacy <strong>of</strong> Hematopoietic Stem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong> forHurler Type 1 MucopolysaccharidosisStefania Delai, Ilaria Visigalli, Letterio S. Politi, Carmela Di Domenico, Emanuela Mrak,Riccardo Brambilla, Federica Cerri, Daniela Ungaro, Merel Stok, Francesca Sanvito,Ubaldo Del Carro, Alessandro Rubinacci, Katherine P. Ponder, Angelo Quattrini, Paola DiNatale, Luigi Naldini, Alessandra Biffi.316 2:45 PMTherapeutic Potential <strong>of</strong> <strong>Gene</strong>tically Modified Neural Stem <strong>Cell</strong>s(NSCs) in a Mouse Model <strong>of</strong> Globoid <strong>Cell</strong>s Leukodystrophy (GLD)Margherita Neri, Alessandra Ricca, Silvia Ungari, Ilaria Visigalli, Alessandra Biffi, Ilaria DiGirolamo, Sabata Martino, Lucia Sergi Sergi, Luigi Naldini, Angela Gritti.317 3:00 PMAdeno-Associated Virus and Tetanus Toxin Fragment C Based Vectorsfor <strong>Gene</strong> <strong>Therapy</strong> in the CNSCarlos Gay-Antaki, John H. Wolfe.318 3:15 PMLineage Specific Lentiviral Mpl Expression Rescues HematopoieticStem <strong>Cell</strong> Defects in Mpl-/- MiceDirk Heckl, Daniel C. Wicke, Johann Meyer, Axel Schambach, Johanna Krause, MatthiasBallmaier, Christopher H. Baum, Ute Modlich.319 3:30 PMSafety <strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong> for ThalassemiaRiccardo Mezzadra, Emanuela Roselli, Claudia Rossi, Francesca Tiboni, GiacomoMandelli, Sarah Marktel, Fulvio Mavilio, Giulietta Maruggi, Marco Andreani, GuidoLucarelli, Giuliana Ferrari.320 3:45 PMLong-Term Phenotypic Correction <strong>of</strong> Skin by Fetal <strong>Gene</strong> Transfer in aLethal Murine Model <strong>of</strong> Epidermolysis BullosaMasayuki Endo, Philip W. Zoltick, Antenata Radu, Jiang Qiujie, Chihiro Matsui, Peter M.Marinkovich, Jouni Uitto, Alan W. Flake.Oral Abstract Session 324Physical Methods <strong>of</strong> Delivery2:00 PM - 4:00 PMRoom: Wilson ABC321 2:00 PMBile Duct Infusion <strong>of</strong> Plasmid DNA Mediated High Level <strong>of</strong> TransgeneExpression in Rat LiverXuan Jiang, Derek Leong, Deng Pan, Hai-Quan Mao.322 2:15 PMImage-Guided, Computer-Controlled Hydrodynamic <strong>Gene</strong> Delivery toLiver Results in Therapeutic Level <strong>of</strong> Human Alpha-1 Antitrypsin inPigs and DogsKenya Kamimura, Takeshi Suda, Guisheng Zhang, Dexi Liu.323 2:30 PMTargeting Magnetic Nanoparticles Impregnated with Adenovirus toStented Arteries Using Uniform-Field Induced Magnetization in theRat Carotid Model <strong>of</strong> RestenosisMichael Chorny, Ilia Fishbein, Richard Adamo, Robert J. Levy.324 2:45 PMUltrasound-Activated Microbubbles for <strong>Gene</strong> Transfer: In VivoEfficiency and Confocal Microscopy Real Time IntracellularInvestigationsAnthony Delalande, Patrick Midoux, Michiel Postema, Chantal Pichon.325 3:00 PMLong-Term <strong>Gene</strong> Expression after Hydrodynamic Delivery <strong>of</strong> SleepingBeauty Transposons to Canine Liver Using Balloon CathetersPerry B. Hackett, Myra Urness, Jason B. Bell, Erik R. Olson, Elena L. Aronovich, R. ScottMcIvor.326 3:15 PMEfficient Microbubble- and Ultrasound-Mediated Plasmid DNATransfer into a Specific Rat Liver Lobe Via a Targeted Injection andAcoustic Exposure Using a Novel Ultrasound SystemShuxian Song, Misty L. Noble, Liping Chen, Samuel S. Sun, Andrew A. Brayman, CarolH. Miao.EXHIBITOR PROSPECTUSfinal program
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