13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010123Abstract DirectorySaturday, May 22, 2010Oral Abstract Session 412Cardiovascular <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>11:00 AM - 1:00 PMRoom: Wilson ABC596 11:00 AMSystemic Delivery <strong>of</strong> AAV2/9 Vectors Improve Cardiac Function inPompe DiseaseDarin J. Falk, Meghan S. Soustek, Cathryn S. Mah, Denise A. Cloutier, Sean A. Germain,Barry J. Byrne.597 11:15 AMCoordinated Co-Expression <strong>of</strong> VEGF 164and PDGF-BB PreventsAberrant Angiogenesis and Accelerates Vascular StabilizationRoberto Gianni-Barrera, Silvia Reginato, Thomas Wolff, Lorenz Gürke, Michael Heberer,Andrea Banfi.598 11:30 AMMitral Valve Disease Is Due to Abnormal Collagen Structure in CanineMucopolysaccharidosis I and VII and Can Be Reduced with NeonatalIntravenous Injection <strong>of</strong> a Gamma Retroviral VectorKatherine P. Ponder, Meg M. Sleeper, Susan Wu, Patricia O’Donnell, Mark E. Haskins.599 11:45 AMAAV9-Mediated proBNP Delivery Improves Diastolic Performance ina Model <strong>of</strong> Spontaneous Hypertensive RatsJason M. Tonne, Jarryd M. Campbell, Diego Bellavia, Elise A. Oehler, Fernando L. Martin,Alessandro Cataliotti, John C. Burnett, Yasuhiro Ikeda.600 12:00 PMIn Vivo Over-Expression <strong>of</strong> a b-Adrenergic Receptor Kinase InhibitorUsing scAAV6-Mediated Molecular Cardiac Surgery with RecirculatingDelivery (MCARD) Enhances Cardiac FunctionJaBaris D. Swain, Michael G. Katz, Anthony Fargnoli, Marina Sumaroka, CatherineTomasulo, Mihail Petrov, James J. Pilla, Hansell H. Stedman, Walter J. Koch, JosephRabinowitz, Charles R. Bridges.601 12:15 PMAAV-Mediated SERCA2a Expression Improves theElectrocardiographic Pr<strong>of</strong>ile <strong>of</strong> Aged mdx MiceJin-Hong Shin, Brian P. Bostick, Yongping Yue, Roger J. Hajjar, Dongsheng Duan.602 12:30 PMRNAi-Mediated Knockdown <strong>of</strong> HMG CoA Reductase In Vitro and InVivo Increases Transgene Expression from Genomic LDLR Mini-<strong>Gene</strong>VectorsOlivia C. Hibbitt, Sarina Agkatsev, Carina Owen, Len Seymour, Keith M. Channon,Richard Wade-Martins.603 12:45 PMSDF-1a-Engineered <strong>Cell</strong>-Based <strong>Therapy</strong> for Diabetic Wound HealingZhao-Jun Liu, Runxia Tian, Weijun An, Ying Zhuge, Yan Li, Hongwei Shao, Bianca Habib,Omaida C. Velazquez.Oral Abstract Session 413Cancer-Oncolytic Viruses II11:00 AM - 1:00 PMRoom: Marriott Ballroom Salons 2 & 3604 11:00 AMHigh Resolution Analysis <strong>of</strong> the Intratumoral Spread <strong>of</strong> anIntravenously Administered Oncolytic VirusShruthi Naik, Rebecca Nace, Elizabeth Hadac, Glen Barber, Stephen J. Russell.605 Abstract Withdrawn191 11:15 AMA Comparative Study <strong>of</strong> Stem <strong>Cell</strong>-Based <strong>Cell</strong> Carrier Systems for theSystemic Delivery <strong>of</strong> an Oncolytic Adenovirus for Antiglioma <strong>Therapy</strong>Matthew A. Tayler, Atique U. Ahmed, Ilya V. Ulasov, Irina Y. Balyasnikova, Maciej S.Lesniak.606 11:30 AMCombination <strong>of</strong> Anti Angiogenic Cancer <strong>Therapy</strong> with SystemicOncolytic Virotherapy To Treat Established TumorsTimothy Kottke, Ge<strong>of</strong>f Hall, Jose Pulido, Rosa Diaz, Jill Thompson, DebabrataMukhopadhyay, Heung Chong, Peter Selby, Matt C<strong>of</strong>fey, Hardev Pandha, John Chester,Alan Melcher, Kevin Harrington, Richard Vile.607 11:45 AMThe Type III Interferon IL-28 Mediates Anti Tumor Efficacy <strong>of</strong>Oncolytic VSV in a Fully Immune Competent ModelPhonphimon Wongthida, Rosa Diaz, Feorillo Galivo, Timothy Kottke, Jill Thompson, JosePulido, Kevin Pavelko, Larry Pease, Alan Melcher, Richard Vile.608 12:00 PMA New Oncolytic Herpes Simplex Virus That Synergizes with SmallMolecule Inhibitors <strong>of</strong> PI3K/Akt To Treat Malignant GliomaRyuichi Kanai, Matthew Gardner, Hiroaki Wakimoto, Robert L. Martuza, Samuel D.Rabkin.609 12:15 PMImproving the Lytic Capacity <strong>of</strong> a Conditionally ReplicativeAdenoviruses by Incorporating Promoter Elements Responsive toTumor Environmental ConditonsDiego L. Viale, Veronica M. Lopez, Eduardo G. Cafferata, David Gould, Yuti Chenajovsky,Osvaldo L. Podhajcer.610 12:30 PMDefining the Role <strong>of</strong> Innate Immune Mechanisms in the Efficacy <strong>of</strong>Oncolytic VSV Virotherapy in a Fully Immune Competent ModelPhonphimon Wongthida, Rosa Diaz, Feorillo Galivo, Timothy Kottke, Jill Thompson, JosePulido, Alan Melcher, Richard Vile.611 12:45 PMDifferential Effects <strong>of</strong> VSV and Cyclophosphamide on NK <strong>Cell</strong>sLeads to Antagonistic Effects on Anti-Tumor <strong>Therapy</strong> <strong>of</strong> a MurineMesothelioma ModelCandice L. Willmon, Jill Thompson, Timothy Kottke, Stephen Albelda, Richard Vile.Saturday, May 22 nd
124<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectorySaturday, May 22 ndSaturday, May 22, 2010Oral Abstract Session 414Hematologic/Immunologic: Clinical, Preclinical and Safety11:00 AM - 1:00 PMRoom: Maryland Suite612 11:00 AMPolyclonal Reconstitution <strong>of</strong> the Hematopoietic System in TwoPatients <strong>of</strong> a WAS Clinical <strong>Gene</strong> <strong>Therapy</strong> TrialAnna Paruzynski, Kaan Boztug, Claudia Ball, Ali Nowrouzi, Christina Lulay, Marie Böhm,Sonja Naundorf, Klaus Kühlke, Rainer Blasczyk, Irina Kondratenko, Laszló Maródi,Hanno Glimm, Christoph Klein, Christ<strong>of</strong> von Kalle, Manfred Schmidt.613 11:15 AMSustained Partial Phenotypic Correction <strong>of</strong> Hemophilia B in the Dogby a microRNA-Regulated Lentiviral VectorAlessio Cantore, Marco Ranzani, Lucia Sergi Sergi, Giuliana Vallanti, Marina Radrizzani,Brian Brown, Eugenio Montini, Timothy Nichols, Luigi Naldini.614 11:30 AMTiming <strong>of</strong> Anti-T <strong>Cell</strong> Immunosuppressive <strong>Therapy</strong> Is Critical for theSafety <strong>of</strong> Liver Mediated AAV2 Transduction in Rhesus MacaquesJonathan D. Finn, Patricia Favaro, J. Fraser Wright, Federico Mingozzi, Katherine A.High, Valder R. Arruda.615 11:45 AMFactor IX-R338L (FIX Padua) as a Novel Alternative for theTreatment <strong>of</strong> Canine Severe Hemophilia BJonathan D. Finn, Paolo Simioni, Nicholas Iacobelli, Shangzhen Zhou, Timothy C.Nichols, Katherine A. High, Valder R. Arruda.616 12:00 PMErythropoeisis Sustained 1 Year by the EPODURE BioPump inPatients with Chronic Kidney Disease: Further Results <strong>of</strong> Phase I/IIPro<strong>of</strong> <strong>of</strong> Concept TrialBaruch S. Stern, Ehud Shoshani, Andrew L. Pearlman, Philip Ng, Allen R. Nissenson,Eithan Galun, Anatole Besarab, Avi Rimler, Haim Goltzmann, Reem Miari, Alex Okun,Haddas Bookay, Tamar Jibly, Javier A. Abrameto, Michal Elhalel.617 12:15 PMLentiviral <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> RAG1 Severe CombinedImmunodeficiency: Risks and Implications for TreatmentNiek P. van Til, Roya Sarwari, Trudi P. Visser, Helen de Boer, Yuedan Li, Julia Hauer,Chantal Lagresle-Peyrou, Olivier Danos, Arnaud Jollet, Fang Zhang, Adrian Thrasher,Barbara Cassani, Monique M. Verstegen, Anna Villa, Marina Cavazzana-Calvo, GerardWagemaker.618 12:30 PMDisruption <strong>of</strong> Glucocorticoid Regulation in Therapeutic Human T <strong>Cell</strong>swith Zinc Finger NucleasesRebecca A. Gardner, Andreas Reik, Carolina Berger, Seitaro Terakura, Philip D. Gregory,Michael C. Jensen, Michael C. Holmes, Stanley R. Riddell.619 12:45 PMLentiviral <strong>Gene</strong> Transfer under Optimized Cytokine ConditionsFacilitates Polyclonal Hematopoiesis <strong>of</strong> Recipient MiceTobias Maetzig, Martijn H. Brugman, Olga S. Kustikova, Zhixiong Li, Melanie Galla, NielsHeinz, Ute Modlich, Bernhard Schiedlmeier, Axel Schambach, Christopher Baum.Oral Abstract Session 415Lung and Respiratory Disease <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>11:00 AM - 12:00 PMRoom: Thurgood Marshall W620 11:00 AM<strong>Gene</strong>ration <strong>of</strong> a Transgenic Gut-Corrected CFTR Knockout FerretModel with Intestinal-Specific Expression <strong>of</strong> Ferret CFTRXiaoming Liu, Xingshen Sun, John T. Fisher, Meihui Luo, Hongshu Shui, Ziying Yan,Yaling Yi, Michael J. Goodheart, Gergely Lukacs, David Meyerholz, John F. Engelhardt.621 11:15 AMPersistent Long Term Lentiviral Correction <strong>of</strong> the CFTR <strong>Gene</strong> Defect inCystic Fibrosis MicePatricia Cmielewski, Donald Anson, David W. Parsons.622 11:30 AMDevelopment <strong>of</strong> Optimized hCFTR <strong>Gene</strong> Sequence and ProlongedExpression Vector for Cystic Fibrosis <strong>Therapy</strong>Linas Padegimas, Karla A. Dines, Samuel J. Adams, Sharon M. Oette, Susannah L. Hyatt,Olena Tyr, Christopher R. Gedeon, Ozge Sesenoglu-Laird, Robert C. Moen, Tomasz H.Kowalczyk, Mark J. Cooper.623 11:45 AMExpression <strong>of</strong> Human Alpha-1 Antitrypsin in Mice and Dogs FollowingAAV6 Vector-Mediated <strong>Gene</strong> Transfer to the LungsChristine L. Halbert, David K. Madtes, Andrew E. Vaughan, Zejing Wang, Rainer Storb,Stephen J. Tapscott, A. Dusty Miller.Oral Abstract Session 416<strong>Gene</strong> Regulation12:15 PM - 1:15 PMRoom: Thurgood Marshall W624 12:15 PMEngineered Zinc Finger Transcription Factors for the Treatment <strong>of</strong>Gain <strong>of</strong> Function MutationsClaudio Mussolino, Daniela Sanges, Ciro Bonetti, Elena Marrocco, Umberto Di Vicino,Valeria Marigo, Alberto Auricchio, Germana Meroni, Enrico M. Surace.625 12:30 PMAAV Vectors for Tissue-Specific, Regulated Expression in Heart, Liverand LungShu-Jen Chen, Maria P. Limberis, Lawrence Bish, Ruben Hovhannisyan, Sheena Saighal,Odella Jno-Charles, H. Lee Sweeney, Arbans Sandhu, Julie Johnston, James M. Wilson.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 127 and 128: 126American Society of Gene & Cell
- Page 129 and 130: 128American 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
Change language