13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010127Abstract DirectorySaturday, May 22, 2010Oral Abstract Session 432 — continuedCancer – Immunotherapy II3:00 PM - 5:00 PMRoom: Marriott Ballroom Salons 2 & 3656 4:00 PMAn A20-Silenced, Stroma-Targeted DC Vaccine Reverses theImmunosuppressive Tumor Microenviroment Resulting in PotentAntitumor ActivityXiao-Tong Song, Xiaoou Zhou, Lisa Rollins, Cliona M. Rooney, Stephen Gottschalk.657 4:15 PMA CD46-Targeting Recombinant Protein that Increases theTherapeutic Efficacy <strong>of</strong> Monoclonal Antibodies for CancerHongjie Wang, Ying Liu, Zong Li, Akseli Hemminki, Andre Lieber.658 4:30 PMTreatment <strong>of</strong> Cancer Patients with Integrin-Targeted OncolyticAdenoviruses Ad5-D24-RGD and Ad5-RGD-D24-GMCSFSari Pesonen, Vincenzo Cerullo, Sophie Escutenaire, Mari Raki, Lotta Kangasniemi, EerikaKarli, Elina Haavisto, Minna Oksanen, Timo Joensuu, Anna Kanerva, Akseli Hemminki.659 4:45 PMEffective Dendritic <strong>Cell</strong> Immuno-<strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> Established ErbB2-Expressing Tumors Using Pentostatin-Based Host Immune Depletionand Subsequent Host Th1/Tc1 <strong>Cell</strong> TransferMiriam Mossoba, Jason Foley, Anu Gangopadhyay, Kris Ylaya, Matthew Winterton,Justin Taylor, Paul Massey, Stephen M. Hewitt, Jeffrey A. Medin, Daniel H. Fowler.Oral Abstract Session 433Oligonucleotide & RNAi Therapeutics II3:00 PM - 5:00 PMRoom: Thurgood Marshall W660 3:00 PMAlleviation <strong>of</strong> Potentially Adverse shRNA Off-Targeting Via AAVVector-Encoded Passenger Strand DecoysStefan Mockenhaupt, Nina Schürmann, Dirk Grimm.661 3:15 PMMultiplexing <strong>of</strong> snoRNAs for HIV-1 InhibitionJane Zhang, John J. Rossi.662 3:30 PMDelivery <strong>of</strong> Chemo-Sensitizing siRNAs to HER2-Positive BreastCancer <strong>Cell</strong>s Using RNA AptamersKristina W. Thiel, William H. Thiel, Xiuying Liu, Justin P. Dassie, Katie R. Stockdale,William M. Rockey, James O. McNamara, II, Paloma H. Giangrande.663 3:45 PMMethods for Isolating <strong>Cell</strong>-Internalizing RNA Aptamers for TargetedsiRNA DeliveryWilliam H. Thiel, Kristina W. Thiel, Justin P. Dassie, Liu Xiuying, Katie R. Stockdale,William M. Rockey, James O. McNamara, II, Paloma H. Giangrande.664 4:00 PMTreatment with the TLR9 Agonist MGN1703 in Patients withMetastatic Solid Tumors – EFFICACY Results <strong>of</strong> a Phase 1 ClinicalStudyMartin R. Weihrauch, Manuel Schmidt, Heike Richly, Bahar Bahar, Michael S. vonBergwelt-Baildon, Ulrich Hacker, Alexander Shimabukuro-Vornhagen, Marina Tschaika,Burghardt Wittig, Max E. Scheulen.665 4:15 PMmiR-7 Inhibits Schwannoma Tumor Growth by Directly Targetingp21-Activated Kinase 1 and Epidermal Growth Factor ReceptorOkay Saydam, Ozlem Senol, Arda Mizrak, Thomas Wurdinger, Anat O. Stemmer-Rachamimov, Anna M. Krichevsky, Xandra O. Breakefield.666 4:30 PMNanoparticles are Effective Vehicles for Systemic Delivery <strong>of</strong>2’OMePS Antisense Oligonucleotides in Exon Skipping-MediatedDystrophin RestorationAlessandra Ferlini, Marina Fabris, Elena Bassi, S<strong>of</strong>ia Falzarano, Daniela Perrone,Francesca Gualandi, Patrizia Sabatelli, Luciano Merlini, Paola Rimessi.667 4:45 PM<strong>Therapy</strong> for Vaso-Occlusion in Sickle <strong>Cell</strong> Disease Includes RNAAptamerAngela D. Burnette, Jun-ichi Nishimura, Milena Batchvarova, Shahid M. Nimjee, RahimaZenadi, Marilyn J. Telen, Bruce A. Sullenger.Oral Abstract Session 434<strong>Cell</strong> Processing and Vector Production3:00 PM - 5:00 PMRoom: Marriott Ballroom Salon 1668 3:00 PMGMP Production <strong>of</strong> Self-Complementary Serotype 8 AAV Vector forTreatment <strong>of</strong> Hemophilia BJames A. Allay, John S. Coleman, Arthur W. Nienhuis, Andrew David<strong>of</strong>f, Amit C.Nathwani, Susan Sleep, John T. Gray.669 3:15 PMProduction <strong>of</strong> Lentiviral Vectors in Suspension <strong>Cell</strong>s UsingRecombinant BaculovirusesHanna P. Lesch, Anna E. Laitinen, Lauri M. Laitinen, Jere T. Pikkarainen, HarithaSamaranayake, Seppo Ylä-Herttuala, Kari J. Airenne.670 3:30 PMIncreased Therapeutic Effect <strong>of</strong> <strong>Cell</strong> Transplantation for WoundHealing in Diabetic Mice by Prolonging the Survival <strong>of</strong> Transplanted<strong>Cell</strong>s Using Adhesamine, a Synthetic Adhesion MoleculeTetsuya Suehara, Makiya Nishikawa, Yuki Takahashi, Sayumi Yamazoe, MotonariUesugi, Yoshinobu Takakura.671 3:45 PMCharacterization <strong>of</strong> Positioning Effect <strong>of</strong> pol III-shRNA TranscriptionUnit in scAAV Vector Genome on the Packaging Efficiency andFunctionality <strong>of</strong> shRNA SilencingJun Xie, Ran He, Qin Su, Guangping Gao.J. Telen, Bruce A. Sullenger.Saturday, May 22 nd
128<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectorySaturday, May 22, 2010Oral Abstract Session 434 — continued<strong>Cell</strong> Processing and Vector Production3:00 PM - 5:00 PMRoom: Marriott Ballroom Salon 1672 4:00 PMRapid Production <strong>of</strong> High Titer Virus Using a Concentrated RetrovirusExpansion DeviceCeidy Sanchez, Gianpietro Dotti, John Wilson, Cliona M. Rooney, Malcolm K. Brenner,Juan F. Vera.673 4:15 PMA Novel Approach to Developing Recombinant AAV Producing <strong>Cell</strong>LinesJingmin Zhou, Katherine A. High, Guang Qu.674 4:30 PMAn Efficient and Complete Platform for the Production and Use <strong>of</strong>Lentiviral VectorsThomas Quinn, Chris Mello, Lily Lee, Mei Fong, Dwayne Bisgrove, Michael Haugwitz,Hiroaki Sagawa.675 4:45 PMDevelopment, Scale-Up and Technology Transfer for Rapid,Automated, cGMP Compliant, Large-Volume Suspension <strong>Cell</strong> BasedProcess for Lentiviral Vector ProductionJames Brady, Linhong Li, Scott R. Witting, Cornell Allen, Rama Shivakumar, Aparna Jasti,Kenneth G. Cornetta, Madhusudan V. Peshwa.678 3:30 PMUpdate <strong>of</strong> Stem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong> Clinical Trial for ADA-Deficiency inJapanMakoto Otsu, Masafumi Yamada, Yoshihiro Maeyama, Miyuki Kida, Satoru Nakajima,Nariaki Toita, Norikazu Hatano, Ryouji Kobayashi, Osamu Tatsuzawa, Junichi Mineno,Masafumi Onodera, Pawan Bali, Michael S. Hershfield, Fabio Candotti, NobuakiKawamura, Yukio Sakiyama, Tadashi Ariga.679 3:45 PMRescue and Long-Term Outcome <strong>of</strong> a Murine Model <strong>of</strong> MethylmalonicAcidemia Following Liver-Directed, AAV-Mediated <strong>Gene</strong> <strong>Therapy</strong>Randy J. Chandler, Nuria Carillo-Carrasco, Suma Chandrasekaran, Charles P. Venditti.680 4:00 PMAAV Vector Mediated Enzyme Replacement <strong>Therapy</strong> <strong>of</strong>Hypophosphatasia (HPP) Model MiceTae Matsumoto, Seiko Yamamoto, Hideo Orimo, Koichi Miyake, Noriko Miyake, SonokoNarisawa, José Luis Millán, Yoshitaka Fukunaga, Takashi Shimada.681 4:15 PMrDNA-AAV Vectors for the Treatment <strong>of</strong> PhenylketonuriaNick Morcinek, Laura M. Roy, Zhongya Wang, Markus Grompe, Cary Harding.682 4:30 PMSafety and Efficiency <strong>of</strong> Site-Specific Integration AAV-rDNA VectorsZhongya Wang, Mark Kay, Markus Grompe.683 4:45 PMFunctional Improvement <strong>of</strong> LGMD Hamsters by AAV-hMG53-Mediated Sarcolemma RepairBo He, Chunping Qiao, Bin Xiao, Juan Li, Zhenhua Yuan, Christina Mayer, Jianjie Ma,Xiao Xiao.Saturday, May 22 ndOral Abstract Session 435<strong>Gene</strong> <strong>Therapy</strong> for Inborn Errors <strong>of</strong> Metabolism3:00 PM - 5:00 PMRoom: Wilson ABC676 3:00 PMEarly Gestational <strong>Gene</strong> Transfer with Lentiviral Vector Driven bya Liver Specific Promoter Targets the Fetal Liver and ImprovesPhenotype in a Murine Model <strong>of</strong> Wilson’s DiseaseJessica L. Roybal, Masayuki Endo, Antoneta Radu, Carlyn Todorow, Philip W. Zoltick,Alan W. Flake.677 3:15 PMA Combination <strong>of</strong> Pharmacological and Liver-Directed <strong>Gene</strong> <strong>Therapy</strong>Results in Long-Term Phenotypic Correction <strong>of</strong> a Mouse Model <strong>of</strong>Argininosuccinic AciduriaPhilippe M. Campeau, Oleg A. Shchelochkov, Sandesh C. Sreenath Nagamani, NicolaBrunetti-Pierri, Donna Palmer, Muralidhar H. Premkumar, Harsha K. Garg, Anilkumar K.Reddy, Yaoping Tang, Nathan S. Bryan, Ayelet Erez, Brendan H. Lee.Oral Abstract Session 436Late Breaking Science3:00 PM - 5:00 PMRoom: Virginia Suite914 3:00 PMPostexposure Protection <strong>of</strong> Nonhuman Primates against a LethalEbola Virus Challenge by RNA InterferenceMarjorie Robbins, Amy CH Lee, Joan B Geisbert, Anna N Honko, Vandana Sood, Joshua CJohnson, Susan de Jong, Iran Tavakoli, Adam Judge, Thomas W Geisbert, Lisa E Hensley,Ian MacLachlan.915 3:15 PMA Clinical Study to Evaluate the Safety and Efficacy <strong>of</strong> pGM169/GL67A Administered to the Nose and Lungs <strong>of</strong> Individuals with CysticFibrosisStephen C Hyde, Jane C Davies, Uta Griesenbach, Tracy Higgins, Eric WFW Alton, AChristopher Boyd, J Alastair Innes, David Porteous, Deborah R Gill.916 3:30 PMLong-Term Safety and Transgene Expression in MonkeysAdministered Lentiviral and AAV Vectors In UteroAF Tarantal, CCI Lee, C Mall, BJ Byrne, DB Kohn.EXHIBITOR PROSPECTUSfinal program
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