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
- Page 1 and 2:
FINAL PROGRAMAmerican Society of Ge
- Page 4:
13 th AnnUAL MEETing | Washington,
- Page 7 and 8:
6American Society of Gene & Cell Th
- Page 12 and 13:
13 th AnnUAL MEETing | Washington,
- Page 20 and 21:
13 th AnnUAL MEETing | Washington,
- Page 22 and 23:
13 th AnnUAL MEETing | Washington,
- Page 24 and 25:
13 th AnnUAL MEETing | Washington,
- Page 26 and 27:
13 th AnnUAL MEETing | Washington,
- Page 28 and 29:
13 th AnnUAL MEETing | Washington,
- Page 30 and 31:
13 th AnnUAL MEETing | Washington,
- Page 32 and 33:
13 th AnnUAL MEETing | Washington,
- Page 34 and 35:
13 th AnnUAL MEETing | Washington,
- Page 36 and 37:
13 th AnnUAL MEETing | Washington,
- Page 38 and 39:
13 th AnnUAL MEETing | Washington,
- Page 40 and 41:
13 th AnnUAL MEETing | Washington,
- Page 42 and 43:
13 th AnnUAL MEETing | Washington,
- Page 44 and 45:
13 th AnnUAL MEETing | Washington,
- Page 46 and 47:
13 th AnnUAL MEETing | Washington,
- Page 48 and 49:
13 th AnnUAL MEETing | Washington,
- Page 50 and 51:
13 th AnnUAL MEETing | Washington,
- Page 52 and 53:
13 th AnnUAL MEETing | Washington,
- Page 54 and 55:
13 th AnnUAL MEETing | Washington,
- Page 56 and 57:
13 th AnnUAL MEETing | Washington,
- Page 58 and 59:
13 th AnnUAL MEETing | Washington,
- Page 60 and 61:
13 th AnnUAL MEETing | Washington,
- Page 62 and 63:
13 th AnnUAL MEETing | Washington,
- Page 64 and 65:
13 th AnnUAL MEETing | Washington,
- Page 66 and 67:
13 th AnnUAL MEETing | Washington,
- Page 68 and 69:
13 th AnnUAL MEETing | Washington,
- Page 70 and 71:
13 th AnnUAL MEETing | Washington,
- Page 72 and 73:
13 th AnnUAL MEETing | Washington,
- Page 74 and 75:
13 th AnnUAL MEETing | Washington,
- Page 76 and 77:
13 th AnnUAL MEETing | Washington,
- Page 78 and 79: 13 th AnnUAL MEETing | Washington,
- Page 80 and 81: 13 th AnnUAL MEETing | Washington,
- Page 82 and 83: 13 th AnnUAL MEETing | Washington,
- Page 84 and 85: 13 th AnnUAL MEETing | Washington,
- Page 86 and 87: 13 th AnnUAL MEETing | Washington,
- Page 88 and 89: 13 th AnnUAL MEETing | Washington,
- Page 90 and 91: 13 th AnnUAL MEETing | Washington,
- Page 92 and 93: 13 th AnnUAL MEETing | Washington,
- Page 94 and 95: 13 th AnnUAL MEETing | Washington,
- Page 96 and 97: 13 th AnnUAL MEETing | Washington,
- Page 98 and 99: 13 th AnnUAL MEETing | Washington,
- Page 100 and 101: 13 th AnnUAL MEETing | Washington,
- Page 102 and 103: 13 th AnnUAL MEETing | Washington,
- Page 104 and 105: 13 th AnnUAL MEETing | Washington,
- Page 106 and 107: 13 th AnnUAL MEETing | Washington,
- Page 108 and 109: 13 th AnnUAL MEETing | Washington,
- Page 110 and 111: 13 th AnnUAL MEETing | Washington,
- Page 112 and 113: 13 th AnnUAL MEETing | Washington,
- Page 114 and 115: 13 th AnnUAL MEETing | Washington,
- Page 116 and 117: 13 th AnnUAL MEETing | Washington,
- Page 118 and 119: 13 th AnnUAL MEETing | Washington,
- Page 120 and 121: 13 th AnnUAL MEETing | Washington,
- Page 122 and 123: 13 th AnnUAL MEETing | Washington,
- Page 124 and 125: 13 th AnnUAL MEETing | Washington,
- Page 127: 126American Society of Gene & Cell
- Page 131 and 132: 130American Society of Gene & Cell
- Page 133 and 134: 132American Society of Gene & Cell
- Page 135 and 136: 134American Society of Gene & Cell
- Page 137 and 138: 136American Society of Gene & Cell
- Page 139 and 140: 138American Society of Gene & Cell
- Page 141 and 142: 140American Society of Gene & Cell
- Page 143 and 144: 142American Society of Gene & Cell
- Page 145 and 146: 144American Society of Gene & Cell
- Page 147 and 148: 146American Society of Gene & Cell
- Page 149 and 150: 148American Society of Gene & Cell
- Page 151 and 152: 150American Society of Gene & Cell
- Page 153 and 154: 152American Society of Gene & Cell
- Page 155 and 156: 154American Society of Gene & Cell
- Page 157 and 158: 156American Society of Gene & Cell
- Page 159 and 160: 158American Society of Gene & Cell
- Page 161 and 162: 160American Society of Gene & Cell
- Page 163 and 164: 162American Society of Gene & Cell
- Page 165 and 166: 164American Society of Gene & Cell
- Page 167 and 168: 166American Society of Gene & Cell
- Page 169 and 170: 168American Society of Gene & Cell
- Page 171 and 172: 170American Society of Gene & Cell
- Page 173 and 174: 172American Society of Gene & Cell
- Page 175 and 176: 174American Society of Gene & Cell
- Page 177 and 178: 176American Society of Gene & Cell
- Page 179 and 180:
178American Society of Gene & Cell
- Page 181 and 182:
180American Society of Gene & Cell
- Page 183 and 184:
182American Society of Gene & Cell
- Page 185 and 186:
184American Society of Gene & Cell
- Page 187 and 188:
186American Society of Gene & Cell