13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010129Abstract DirectorySaturday, May 22, 2010Oral Abstract Session 436 — continuedLate Breaking Science3:00 PM - 5:00 PMRoom: Virginia Suite917 3:45 PMrAAV- Mediated Therapeutic Silencing <strong>of</strong> miR-122 Leads to Rapid andSignificant Reduction <strong>of</strong> LDL in DLDR-/-/Apobec1A-/- MiceJun Xie, Jennifer Broderick, Qing Xie, Hongwei Zhang, Ran He, Chengjian Li, Qin Su, LiZhong, Terence R. Flotte, Phillip D. Zamore, Guangping Gao.918 4:00 PMHyperactive Transposons for <strong>Gene</strong>tic Modification <strong>of</strong> InducedPluripotent and Adult Stem <strong>Cell</strong>s: A Novel Non-Viral Paradigm forCoaxed DifferentiationEyayu Belay, Janka Mátrai, Abel Acosta-Sanchez, Ling Ma, Mattia Quattrocelli, LajosMátés, Pau Sancho-Bru, Martine Geraerts, Bing Yan, Joris Vermeesch, Ermira Samara-Kuko, Zoltán Ivics, Catherine Verfaillie, Maurillio Sampaolesi, Zsuzsanna Izsvák, ThierryVandenDriessche, Marinee K.L. Chuah.919 4:15 PMPromoting the Migration and Integration <strong>of</strong> Schwann <strong>Cell</strong>s afterTransplantation into Lesioned Spinal Cord by Lentiviral MediatedExpression <strong>of</strong> Polysialic AcidJuan Luo, Xuenong Bo, Dongsheng Wu, John Yeh, Peter M Richardson, Yi Zhang.920 4:30 PMRescue <strong>of</strong> the Spinal Muscular Atrophy Phenotype in a Mouse Modelby Early Postnatal Delivery <strong>of</strong> SMNKevin D Foust, Xueyong Wang, Vicki L McGovern, Lyndsey Braun, Adam K Bevan,Amanda M Haidet, Thanh T Le, Pablo R Morales, Mark M Rich, Arthur H M Burghes,Brian K Kaspar.921 4:45 PMAptamer-Mediated Delivery <strong>of</strong> siRNAs and miRNA-Based siRNATriggers to the Nucleus <strong>of</strong> Multiple Cancer <strong>Cell</strong> TypesJonathan Kotula, Bruce Sullenger.Poster Session III6:00 PM - 8:00 PMRoom: Exhibit Hall CRNA Virus Vectors II684Insights on the Mechanisms <strong>of</strong> Measles Virus Entry into <strong>Cell</strong>sMatthew K. Ennis, Stephen J. Russell, David Dingli.685The Principal Function <strong>of</strong> the Lentiviral Central DNA Flap Is DefenseAgainst APOBEC3 Protein RestrictionChunling Hu, Dyana T. Saenz, Hind Fadel, Mary E. Peretz, William Walker, Eric M.Poeschla.686In Vivo Immunization with Integration Deficient Lentiviral VectorsBiliang Hu, Pin Wang.687“Same Day” Ex Vivo Regional <strong>Gene</strong> <strong>Therapy</strong>: A Novel Approach ToEnhance Bone RepairMandeep S. Virk, O. Sugiyama, Sang H. Park, Jay R. Lieberman.688The R Peptide Sequence <strong>of</strong> XMRV Envelope Glycoprotein Determinedthe Pseudotyping Specificity with HIV-1 and MLV VectorsToshie Sakuma, Suk See De Ravin, Harry L. Malech, Yasuhiro Ikeda.689Virus-Receptor Mediated Transduction <strong>of</strong> Dendritic <strong>Cell</strong>s among RossRiver Virus and Semliki Forest Virus Pseudotyped LentivectorsSteven Froelich, April Tai, Katie Stroup, Adnan Zubair, Pin Wang.690Effect <strong>of</strong> TLX1 Homeobox <strong>Gene</strong> Expression on InsertionalImmortalization by an Insulated Self-Inactivating Retroviral VectorLynnsey A. Zweier-Renn, Teresa S. Hawley, Ali Ramezani, Irene Riz, Robert G. Hawley.691Transcriptional Pulsing <strong>of</strong> Retroviral Transgenes in Mouse EmbryonicStem <strong>Cell</strong>sMandy Y. M. Lo, Akitsu Hotta, Sylvie Rival-Gervier, James Ellis.692The <strong>Cell</strong>ular Fibronectin Matrix Is a Key Factor for the Attachment<strong>of</strong> VSV-G Pseudotyped Vector Particles and Its <strong>Cell</strong> Type SpecificComplexity Provides a Mechanism for Delayed <strong>Cell</strong> EntryJosha A. Woodward, Amy M. Skinner, Peter Kurre.693Targeting <strong>of</strong> Lentiviral Vectors and Positioning <strong>of</strong> Transduced <strong>Cell</strong>s byMagnetic Nanoparticles and Magnetic FieldKatrin Zimmermann, Christina Trueck, Olga Mykhaylyk, Ulrich M. Becher, BernhardGleich, Daniela Wenzel, Bernd Fleischmann, Christian Plank, Alexander Pfeifer.694Pseudotyped Hepatitis C Virus Particle Could Be a Liver Target Bio-MissileXiaojing Lin, Yonghui Zhang, Yue Wang.695Regulation <strong>of</strong> Cytokine Expression by Lentivirus-Encoded RNAAntagomirsRasmus O. Bak, Line B. Petersen, Brian Moldt, Karin Stenderup, Cecilia Rosada, LarsAagaard, Tomas N. Dam, Jacob G. Mikkelsen.696Effects <strong>of</strong> Replicators on Retroviral Variegation and ExtinctionMeihong Liu, Maribeth V. Eiden.Saturday, May 22 nd
130<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, 2010Poster Session III — continued6:00 PM - 8:00 PMRoom: Exhibit Hall CAAV Vectors III697Late Gestation Intrauterine <strong>Gene</strong> Transfer <strong>of</strong> AAV9 in Non-HumanPrimates Preferentially Transduces the Central Nervous System,Liver, Skeletal and Cardiac MusculatureC. N. Mattar, N. Johana, A. Fisk, A. Biswas, J. Lu, M. H. Tan, L. G. Tan, A. Rahim, S.Waddington, M. Choolani, J. Chan.698Integrin-Linked Endocytosis Directs AAV2 and AAV1 to a HighlyEfficient Transduction PathwayPaul M. Kaminsky, Nicholas W. Keiser, John F. Engelhardt.699Computationally-Guided Rational Design <strong>of</strong> Tropism-Modified AAVVectorsAdam S. Davis, William C. Ray, Thais Federici, Nicholas M. Boulis, Jeffrey S. Bartlett.700Identification <strong>of</strong> Human Hepatocyte Growth Factor Receptor as a<strong>Cell</strong>ular Receptor/Co-Receptor for AAV3Chen Ling, Yuan Lu, Jasmine K. Kalsi, Giridhara R. Jayandharan, Binbin Cheng, SamanthaW. Y. Gee, Katherine E. McGoogan, Li Zhong, Arun Srivastava.701Rapid Transgene Expression and Long-Term Phenotypic Correction<strong>of</strong> a Lethal Mouse Model <strong>of</strong> Methylmalonic Acidemia Using rAAV9-Mediated <strong>Gene</strong> DeliveryJulien S. Senac, Randy J. Chandler, Charles P. Venditti.702Widespread Transduction <strong>of</strong> Brain Parenchyma after IntraventricularInjection <strong>of</strong> AAV9Annagiusi Gargiulo, Enrico M. Surace.703AAV1.9-3 Exhibiting Significantly Impaired Infectivity andSubstantially Delayed Blood Clearance, Is a Novel DetargetedPlatform <strong>of</strong> AAV Random Peptide Display LibrariesKei Adachi, Nicole M. Kotchey, Kimimasa Tobita, Hiroyuki Nakai.704Development <strong>of</strong> the D[+]-Sequence-Deleted ssAAV2 Vectors:Enhanced Transgene Expression In Vitro and In VivoChen Ling, Giridhara R. Jayandharan, Baozheng Li, Yuan Lu, Wenqin Ma, Arun Srivastava.705Influence <strong>of</strong> Murine Parvovirus on Adeno-Associated Virus Vector-Related Expression in Murine ModelsSara A. Collins, Gerald C. O’Sullivan, Patrick T. Harrison, Martina F. Scallan, MarkTangney.706Involvement <strong>of</strong> the Glucocorticoid Receptor Signaling in AAV2 Vector-Mediated Transgene ExpressionYuan Lu, Chen Ling, Giridhara R. Jayandharan, Angela E. Rivers, Arun Srivastava.707AAV9-Mediated Catalase Expression in Mitochondria ImprovesExercise Performance in mdx MiceDejia Li, Dongsheng Duan.708Development <strong>of</strong> a Liver Specific Tet-On Inducible System for AAVVectorsLucia Vanrell, Laura Blanco, Lilianne Tenenbaum, Jesus Prieto, Gloria González-Aseguinolaza.709Intracortical Delivery <strong>of</strong> an Adeno-Associated Viral Vector Expressingthe Glutamate Transporter (GLT1) Decreases Stroke-InducedExtracellular Glutamate Overflow and Cerebral Infarction afterTransient Middle Cerebral Artery Occlusion in RatsBrandon K. Harvey, Mikko T. Airavaara, Jason Hinzman, Matthew J. Chiocco, Hui Shen,Douglas B. Howard, Greg A. Gerhardt, Barry J. H<strong>of</strong>fer, Yun Wang.710The Potential Role <strong>of</strong> Chromatin Structure in Adeno-Associated VirusType 2 Locus-Specific Integration at AAVS1Victor J. McAlister, Roland A. Owens.711Augmentation <strong>of</strong> Traditional Ad/AAV Helper Plasmids with HPV E1Increases rAAV DNA Replication and Virus Production in 293 <strong>Cell</strong>sSarmistha Bandyopadhyay, Maohua Cao, Yong Liu, Paul L. Hermonat.Adenovirus and Other DNA Virus Vectors III712Hexon-Modifications <strong>of</strong> hAd5 Based Helper-Virus for High-CapacityAdenoviral Vector Production and Limitations Imposed by HVR-Exchanges Utilizing Serotype SwitchMartin A. Hausl, Zsolt Ruzsics, Anja Ehrhardt.713Use <strong>of</strong> <strong>Cell</strong>-Penetrating Peptides To Induce a Bystander EffectYing Shen, William Yu, John G. Hay, Harald Sauth<strong>of</strong>f.714Highly Sensitive Detection <strong>of</strong> Prostate Cancer <strong>Cell</strong>s In Vivo by a DualAdenoviral TSTA Trans-Amplification SystemMakoto Sato, Frédéric Pouliot, Steve Huyn, Lily Wu.715Coagulation Factor X-Mediated Adenoviral Transduction Is Dependenton Heparan Sulfate Proteoglycan SulfationAngela C. Bradshaw, Alan L. Parker, Stuart A. Nicklin, John H. McVey, Andrew H. Baker.EXHIBITOR PROSPECTUSfinal program
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