13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010111Abstract DirectoryFriday, May 21, 2010Poster Session II— continued6:30 PM - 8:30 PMRoom: Exhibit Hall C & B-SouthChemical and Molecular Conjugates II396Intracellular Trafficking and Decondensation Kinetics <strong>of</strong> ChitosanpDNAPolyplexesMarc Thibault, Surendra Nimesh, Lavertu Marc, Buschmann Michael.397Dual Function Effect on PEI-Mediated <strong>Gene</strong> Delivery by DiphtheriaToxin-Derived Ligand and Fusogenic DomainsTakeshi Nagasaki, Shinji Kakimoto, Hideki Azuma.398Tumour <strong>Therapy</strong> with Listeria Monocytogenes Vector-Mediated DNADeliveryJan P. Van Pijkeren, David Morrissey, Michelle Cronin, Simon Rajendran, Gerald C.O’Sullivan, Cormac G. M. Gahan, Mark Tangney.399Efficient <strong>Gene</strong> Transfer to Umbilical Cord Blood-Derived MesenchymalStem <strong>Cell</strong>s Mediated by MR Imageable Inorganic-Organic HybridConjugateYong Sook Kim, In-Kyu Park, Jin Sook Kwon, Myung Ho Jeong, Youngkeun Ahn.400Histidine-Rich Amphipathic Peptides Promote Efficient Delivery <strong>of</strong>Nucleic Acids into Mammalian <strong>Cell</strong>sAntoine Kichler, A. James Mason, Christian Leborgne, Daniel Scherman, BurkhardBechinger.401MHC Class-I Mediated Antigen Presentation by a Non-Viral T-MENDVector Expressing a High Luciferase Activity in Murine DerivedDendritic <strong>Cell</strong>s (JAWS-II)Sharif M. Shaheen, Hidetaka Akita, Takashi Nakamura, Shiroh Futaki, Atsushi Yamashita,Ryo Katoono, Nobuhiko Yui, Hideyoshi Harashima.402Evaluation <strong>of</strong> Polymer-DNA Nanoparticles for <strong>Gene</strong> Transfer toCardiac MusclesXiaohong Qiu, Lepeng Zeng, Jon Urban, Alena Nikolskaya, Sarah Audet.403Chemical Manipulation <strong>of</strong> AAV Tissue TropismEric D. Horowitz, Jana L. Phillips, Aravind Asokan.404Versatile Self-Assembling Nanoparticles for Enhanced siRNA Deliveryinto Mammalian <strong>Cell</strong>sGulam H. Dar, Madhusudhana R. Nalam, Vijaya Gopal.405Novel L-Lysine Reducible Copolymers for Efficient Nonviral <strong>Gene</strong>DeliveryMohamed M. Ismail Nounou, Samuel Chung, Kalyopy Emmanouil, Thu Pham, ZhuoyuanLu, Malavosklish Bikram.406JL1-Directed Small Interfering RNA Delivery Using Anti-JL1 Immuno-Sendai Viroplexes to T-Leukemic <strong>Cell</strong>sJung Seok Kim, Yeon Jyung Lee, Jin Ee Baek, Sang Il Park, Hwa Yeon Jeong, HongSung Kim, Keun Sik Kim, Sang Soon Yoon, Kyeong Cheon Jung, Hyung Geun Song,Yong Serk Park.407Antibody-Directed Drug Delivery Using Protein A-Based CarriersNahyun Kim, Hong-Seok Ban, Priti Kumar, Sang-Kyung Lee.<strong>Gene</strong>tic and Metabolic Diseases <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II408Lentiviral Vector-Transduced Hematopoietic Stem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong>for CystinosisBrian A. Yeagy, Frank Harrison, Matthew V. Tadlock, Daniel R. Salomon, StephanieCherqui.409Creation and Rescue <strong>of</strong> a Severe Hyperammonaemic Phenotype inOrnithine Transcarbamylase Deficient spf ash Mice Using rAAV andshRNASharon C. Cunningham, Cindy Y. Kok, Allison P. Dane, Kevin H. Carpenter, Ian E.Alexander.410Long Term Correction <strong>of</strong> Biochemical and Neurological Abnormalities<strong>of</strong> Metachromatic Leukodystrophy Model Mice by Systemic NeonatalInjection <strong>of</strong> Serotype 9 AAV VectorNoriko Miyake, Koichi Miyake, Nagisa Asakawa, Motoko Yamamoto, Takashi Shimada.411Neural Stem <strong>Cell</strong>s as a Therapeutic for Krabbe’s DiseaseMette Flaat, Cynthia Ripoll, Jimin Yu, Jessica Eiermann, Jeanne Fisher-Perkins, BrittniScruggs, Helen Paige Leonard, Amy Lin, Marjorie McCants, Michelle Eagle, WilliamWimley, Bruce A. Bunnell.412AAV8 Mediated Expression <strong>of</strong> LDLR AmelioratesHypercholesterolemia and Regresses Pre-Existing Atherosclerosis ina Humanized Mouse Model <strong>of</strong> Familial HypercholesterolemiaSadik H. Kassim, Luk H. Vandenberghe, Christian Hinderer, Dawn Marchadier, PeterL. Bell, Hui Li, Debra Cromley, Aisha Wilson, Hongwei Yu, James M. Wilson, Daniel J.Rader.413Widespread Enzymatic Correction <strong>of</strong> CNS Tissues by a SingleIntracerebral Injection <strong>of</strong> Therapeutic Lentiviral Vector inLeukodystrophy Mouse ModelsAnnalisa Lattanzi, Claudio Maderna, Margherita Neri, Ilaria Di Girolamo, Sabata Martino,Aldo Orlacchio, Mario Amendola, Luigi Naldini, Angela Gritti.Friday, May 21 st
112<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-South<strong>Gene</strong>tic and Metabolic Diseases <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II414<strong>Gene</strong> <strong>Therapy</strong> Ex Vivo in the ADA-Deficient Mouse: IncreasedSurvival and Efficacy with Short-Term Enzyme Replacement <strong>Therapy</strong>Post <strong>Gene</strong> <strong>Therapy</strong>Denise A. Carbonaro, Xiangyang Jin, Donald B. Kohn.415Towards Targeted Transgene Integration in Human Hematopoietic andInduced Pluripotent Stem <strong>Cell</strong>sHongjie Wang, Ying Liu, Michael C. Holmes, Philip D. Gregory, Andre Lieber.416Optimization <strong>of</strong> the Expression Cassette Using an AAV2/8 BasedVector for the Treatment <strong>of</strong> Ornithine Transcarbamylase DeficiencyLili Wang, Huan Wang, Hiroki Morizono, Peter L. Bell, David Jones, Mark L. Batshaw,James M. Wilson.417<strong>Gene</strong>ration <strong>of</strong> Early AMD Mouse Model by Induction <strong>of</strong> OxidativeStressSoojung Gibson, Jian W. Liu, Yun Le, Wiliam W. Hauswirth, Alfred S. Lewin.418Increased Expression <strong>of</strong> Human Glucocerebrosidase Activity in BoneMarrow <strong>of</strong> Gaucher Mice Following Macrophage Mediated <strong>Gene</strong><strong>Therapy</strong>Arlene T. Lim, Edward I Ginns.419<strong>Cell</strong> Sheet Engineering toward Bioengineering <strong>of</strong> Functional Neo-IsletsTakahiro Saito, Kazuo Ohashi, Hir<strong>of</strong>umi Shimizu, Rie Utoh, Kazuya Ise, MasayukiYamato, Teruo Okano, Mitsukazu Gotoh.420Liver-Specific Over-Expression <strong>of</strong> PGC-1a Does Not Induce a DiabeticPhenotypeLuke Roode, Chunping Qiao, Ruhang Tang, Jiangjang Jiang, Juan Li, Xiao Xiao.421Promoter Selection for AAV Vector-Mediated Factor IX Expression inSkeletal MuscleAkihiro Kume, Hiroya Yagi, Hiroaki Mizukami, Masashi Urabe, Tomonori Tsukahara, AkiraIshiwata, Jun Mimuro, Seiji Madoiwa, Tsukasa Ohmori, Yoichi Sakata, Keiya Ozawa.422Development <strong>of</strong> HIV-1 Based Lentiviral Vectors Expressing Pre-Trans-Splicing Molecules (PTMs) for Liver-Directed TherapiesMadaiah Puttaraju, S. Gary Mansfield, Jun Wang, Nikolay Korokhov, Jenice D’Costa,Ziping C. Chen, Katherine Radd, Gerard J. McGarrity, Laurent M. Humeau.764 (Moved from Poster Session III)Microporous Polymer Scaffolds for Islet TransplantationRomie F. Gibly, Lonnie D. Shea.Cardiovascular <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>423AAV-Mediated Intramyocardial VEGF-B167 Expression Exerts aPotent Cardioprotective Effect in Small and Large Animal Models <strong>of</strong>Myocardial Infarction and Heart FailureLorena Zentilin, Vincenzo Lionetti, Uday Puligadda, Silvia Moimas, Serena Zacchigna,Martino Pepe, Mohammed Mamdani, Khaled Qanud, Xiabin Xu, Thomas H. Hintze,Fabio A. Recchia, Mauro Giacca.424Osteopontin Enhances the Angiogenic Potential <strong>of</strong> EndothelialProgenitor <strong>Cell</strong>sErin E. Vaughan, Angela Duffy, Timothy O’Brien.425Arginine and Tetrahydrobiopterin Synergistically Potentiate theAntirestenotic Effect <strong>of</strong> Vascular <strong>Gene</strong> <strong>Therapy</strong> with Inducible NitricOxide SynthaseIlia Fishbein, Peter Sobolewki, Richard F. Adamo, Ivan S. Alferiev, Ariel Fishbein, MarinaBakay, Michael Chorny, Robert J. Levy.426Intra-Myocardial Administration <strong>of</strong> ACRX-100 Promotes CardiacBenefit and Improves Angiogenesis in a Porcine Model <strong>of</strong> HeartFailureJoseph M. Pastore, Timothy J. Miller, Rahul A. Aras, Marc S. Penn.427Strategies for Promoting Survival <strong>of</strong> Transplanted Mesenchymal Stem<strong>Cell</strong>s in a Myocardial Infarction ModelLisa McGinley, Daniel O’Toole, Aoife Duffy, Xizhe Chen, Alessia Stocca, Jill McMahon,Timothy O’Brien.428Systemic Delivery <strong>of</strong> Interleukin-10 by an AAV Vector AttenuatesObesity-Related Glomerulopathy in Zucker Fatty RatsManabu Ogura, Masashi Urabe, Akira Onishi, Takayuki Ito, Tomonori Tsukahara, HiroakiMizukami, Akihiro Kume, Eiji Kusano, Keiya Ozawa.429AAV/B7H1-<strong>Gene</strong> Delivery Lowers Th1 Cytotoxic T LymphocyteResponse In Vitro and Atherogenesis In VivoMaohua Cao, Junaid A. Khan, Yong Liu, Paul L. Hermonat.430Dose-Dependent Angiogenesis by Human and Murine VEGF IsSpecies-SpecificEdin Mujagic, Roberto Gianni-Barrera, Marianna Trani, Lorenz Gürke, Michael Heberer,Thomas Wolff, Andrea Banfi.431AAV-Sema3A-Mediated Recruitment <strong>of</strong> Neuropilin-1-ExpressingMononuclear <strong>Cell</strong>s (NEMs) Contributes to Vessel Stabilization andNormalizationAlessandro Carrer, Serena Zacchigna, Silvia Moimas, Lucia Pattarini, Lorena Zentilin,Mauro Giacca.EXHIBITOR PROSPECTUSfinal program
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