13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010119Abstract DirectoryFriday, May 21, 2010Poster Session II — continued6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-SouthImmunologic & Host Responses in <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>538Immunological Effects <strong>of</strong> Oral and Intravenous Cyclophosphamide inCancer Patients Treated with Oncolytic AdenovirusesVincenzo Cerullo, Sophie Escutenaire, Sari Pesonen, Iulia Diaconu, Liisa Sirrka, AnnaKanerva, Lotta Kangasniemi, Elina Haavisto, Minna Oksanen, Tiimo Joensuu, A. Karioja-Kallio, Petteri Arstila, Akseli Hemminki.539The Role <strong>of</strong> TGF-b and IL-10 in the Development <strong>of</strong> ImmuneTolerance Following Hepatic <strong>Gene</strong> <strong>Therapy</strong>Brad E. H<strong>of</strong>fman, Ashley T. Martino, Brandon K. Sack, Roland W. Herzog.540Murine Hepatocytes Transduced with Y-F Mutant Capsid Are MoreResistant to Killing by Capsid Specific CD8+ T <strong>Cell</strong>sAshley Martino, David M. Markusic, Arun Srivastava, Roland W. Herzog.541 Abstract Withdrawn542The Innate Immune Response to Intravenously Injected AdenoviralVectors: Role <strong>of</strong> Endosomal Escape in MAP Kinase Activation and theCytokine/Chemokine ResponseJeffrey S. Smith, Zhili Xu, Jie Tian, Donna J. Palmer, Philip Ng, Andrew P. Byrnes.543AAV8 Vectors Evade CTL Mediated Destruction by Failing To Activatea Potent Inflammatory ResponseSuryanarayan Somanathan, Ekaterina Breous, Peter L. Bell, James M. Wilson.544Robust and Sustained Factor IX Expression by IV Administration <strong>of</strong>AAV8-Based Vectors in MacaquesHiroaki Mizukami, Jun Mimuro, Akira Ishiwata, Hiroya Yagi, Tsukasa Ohmori, SeijiMadoiwa, Tomonori Tsukahara, Masashi Urabe, Akihiro Kume, Yoichi Sakata, KeiyaOzawa.545Induction <strong>of</strong> Transgene Immunological Tolerance by a Single Injection<strong>of</strong> miR-142-Regulated Integrase Defective Lentiviral VectorAndrea Annoni, Alessio Cantore, Lucia Sergi Sergi, Luigi Naldini, Maria Grazia Roncarolo.546T <strong>Cell</strong> Hyperactivity in Cmah Null Mice: A Pre-Clinical rAAVImmunotoxicity Model?George Buchlis, Federico Mingozzi, Daniel J. Hui, Harre Downey, Virginia Haurigot,Shangzhen Zhou, Ajit Varki, Katherine A. High.547Complement-Mediated Clearance <strong>of</strong> AAV by Tissue ResidentMacrophages Limits Transduction <strong>of</strong> Target <strong>Cell</strong>sChelsea M. Bolyard, Jeffrey S. Bartlett.548Overcoming Anti-AAV Neutralizing Antibodies with AlternateSerotype and Pharmacological StrategiesFederico Mingozzi, Margriet J. Vervoordeldonk, Yifeng Chen, Shyrie Edmonson, RogierThurlings, Katherine High, Paul P. Tak.549Switching the Fiber Knob <strong>of</strong> Oncolytic Adenoviruses To AvoidNeutralizing Antibodies in Human Cancer PatientsMari Raki, Iulia Diaconu, Merja Sarkioja, Sophie Escutenaire, Lotta Kangasniemi, AnnaKanerva, Vincenzo Cerullo, Timo Joensuu, Sari Pesonen, Akseli Hemminki.550Depletion <strong>of</strong> B <strong>Cell</strong>s by Anti-CD20 Partially Regulates Anti-FVIIIAntibody Production in the Nonviral <strong>Gene</strong> <strong>Therapy</strong> ModelPeiqing Ye, Baowei Peng, Marilyn Kehry, David J. Rawlings, Carol H. Miao.551Sheep Harbor Naturally-Occurring Antibodies to Human AAV: A NewLarge Animal Model for AAV ImmunologyJoseph Tellez, Jonathan D. Finn, Nick Tschernia, Graça Almeida-Porada, Valder R. Arruda,Christopher D. Porada.552Effects <strong>of</strong> Various TLR/Inflammation Pathways on Humoral and<strong>Cell</strong>ular Immune Response to rAAV1 in MiceMuriel Sudres, Virginie Vasseur, Séverine Ciré, Bernhard Ryffel, Anne Galy.553Monitoring and Suppression <strong>of</strong> Immune Responses to AAV-Mediated<strong>Gene</strong> <strong>Therapy</strong> in Canine MuscleZejing Wang, Rainer Storb, Donghoon Lee, Martin Kushmeric, Baocheng Chu, JefferyChamberlain, Stainly Riddell, Stephen Tapscott.554Prevalence <strong>of</strong> Neutralizing Antibodies Against Adeno-AssociatedVirus 2, 8 and 9 in Non-Human Primate ColoniesHiroya Yagi, Hiroaki Mizukami, Tomonori Tsukahara, Masashi Urabe, Hiromi Hamada,Akihiro Kume, Hiroyuki Yoshikawa, Keiya Ozawa.Oligonucleotide & RNAi Therapeutics II555Dual Function BAFF Receptor Aptamers Block BAFF1 Ligand InducedProliferation and Deliver siRNAS to NHL <strong>Cell</strong>sKatrin Tiemann, Stephen J. Forman, John J. Rossi, Jiehua Zhou.556Efficient Delivery <strong>of</strong> siRNAs into Primary <strong>Cell</strong>s In Vitro andSystemically In Vivo Using Chemically Modified PepFect PeptidesSamir El Andaloussi, Edvard C. I. Smith, Ülo Langel.Friday, May 21 st
120<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, 2010Poster Session II — continued6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-SouthOligonucleotide & RNAi Therapeutics II557Reduction <strong>of</strong> Mutant Huntingtin by Antisense OligonucleotidesAmeliorates Phenotypic Deficits in YAC128 Mouse Model <strong>of</strong>Huntington’s DiseaseJonathan A. Fidler, Lisa M. Stanek, Wendy Yang, Amy M. Richards, Edward Wancewicz,Curt Mazur, <strong>Gene</strong> Hung, C. Frank Bennett, Seng H. Cheng, Lamya S. Shihabuddin.558Antibody-Mediated Targeting <strong>of</strong> pH-Responsive NanoparticlesFacilitates siRNA Delivery to Cancer <strong>Cell</strong>sMaria C. Palanca-Wessels, Richelle Cutler-Strom, Anthony C. Convertine, Fan Lee,Michael F. Press, Scott E. James, Patrick S. Stayton, Oliver W. Press.559Adenoviral shRNA-Mediated Knockdown <strong>of</strong> TNFR1(P55) EitherLocally in the Knee Joint or Systemically Ameliorates Collagen-Induced ArthritisOnno J. Arntz, Jeroen Geurts, Sharon Veenbergen, Miranda B. Bennink, Wim B. van denBerg, Fons A. J. van de Loo.560Specific and Efficacious RNA Aptamer Against Hepatic Metastasis <strong>of</strong>Colon CancerSeong-Wook Lee, Young Ju Lee, Seung Ryul Han, Min jung Kim, Hanna Cho, Won IlLee, Jin-Sook Jeong.561Development <strong>of</strong> Aptamer Therapeutics Against <strong>Cell</strong> Surface Receptorsfor Lymphoma and Auto-Immune DiseasesElizabeth D. Pratico, David Boczkowski, Smita Nair, Bruce A. Sullenger.562A Microscopic Study <strong>of</strong> the Intracellular Fate <strong>of</strong> Cationic MediatedsiRNA DeliveryMeysam Abbasi, Rakesh Bhatnagar, Hasan Uludag.563Deriving Effective Anti-HIV Guide Sequences from Enhanced PrimarymicroRNA Secondary StructuresFiona van den Berg, Patrick Arbuthnot, Marc S. Weinberg.564“Bifunctional” shRNA Strategy for Cancer <strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong>Donald D. Rao, Zhaohui Wang, Padmasini Kumar, Yang Yu, Shihe Liu, Chris M. Jay,Nancy S. Templeton, Courtney Haddock, John Vorhies, Alex W. Tong, Phillip B. Maples,Neil Senzer, F. C. Brunicardi, John Nemunaitis.565Feasibility and Effectiveness <strong>of</strong> Exon 51 skipping in Human-Like mdxMutationYoshitsugu Aoki, Toshifumi Yokota, Takashi Saito, Akinori Nakamura, Tetsuya Nagata,Hitoshi Okazawa, Shin’ichi Takeda.566The Effect <strong>of</strong> RNA Interfering a1,3GT and NF-kB in CardiacXenotransplantationFeng Qi, Chengyu Lv, Yachuan Gu, Xianghui He, Liwei Zhu.567Inhibition <strong>of</strong> Breast Cancer <strong>Cell</strong> Growth Via Silencing IKKeBin Qin, Ravi S. Shukla, Kun Cheng.568Cationic Polymer Mediated siRNA Delivery for P-glycoprotein (P-gp)Down-Regulation in Tumor <strong>Cell</strong>sMeysam Abbasi, Afsaneh Lavasanifar, Michael Weinfeld, Hasan Uludag.569<strong>Gene</strong> Editing Induces Double-Stranded Breakage in Mammalian <strong>Cell</strong>sMelissa A. Bonner, Eric B. Kmiec.<strong>Cell</strong> Processing and Vector Production II570Development <strong>of</strong> a MDCK <strong>Cell</strong> Line for the Manufacturing <strong>of</strong> CanineAdenovirus Type 2 (CAV-2) VectorsVirgínia Santiago, Eric Kremer, Paula M. Alves, Ana S. Coroadinha.571Production <strong>of</strong> a rAAV1-CB-hAAT Human Clinical Trial Lot by rHSVCo-Infection <strong>of</strong> Suspension BHK <strong>Cell</strong>sDavid R. Knop, Brad Grimm, Lijun Wang, Darby L. Thomas, Guo-jie Ye.572RNA Based Plasmid Selection System for Antibiotic-Free PlasmidDNA Vector ProductionAaron E. Carnes, Jeremy M. Luke, Justin M. Vincent, Clague P. Hodgson, James A.Williams.573Robust Recombinase-Free System Production <strong>of</strong> Helper-DependentAdenoviral Vectors with Negligible Contamination by DelayedPackaging ProcessDan Cots, Raul Alba, Patrick Hearing, Eric J. Kremer, Maria Mercedes Segura, AssumpcióBosch, Miguel Chillón.574Removal <strong>of</strong> Clinically Relevant Contaminants from cGMP GradePreparations <strong>of</strong> Plasmid DNA: Implications for Non-Viral <strong>Gene</strong><strong>Therapy</strong> and DNA-Based VaccinesNancy S. Templeton, Pervez Firozi, Wei Zhang, Li Chen, Florante A. Quiocho, Kim C.Worley, Phillip B. Maples, John Nemunaitis.EXHIBITOR PROSPECTUSfinal program
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