FINAL PROGR AM - American Society of Gene & Cell Therapy

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AMERICAN SOCIETY OF GENE THERAPY • Final Program 81 ABSTRACTS FRIDAY, JUNE 3 585 A Water-Soluble Lipoploymer Gene Carrier to Deliver a Bicistronic Plasmid Conveying IL-2 and Soluble Flk-1 for the Treatment of Renal Cell Carcinoma Tumors James W. Yockman, Won Jong Kim, Sung Wan Kim 586 Novel Lipidic Vectors for Targeted Delivery of Functional Oligonucleotides to Pulmonary Endothelium Annette S. Wilson, Stephen J. Kennel, Leaf Huang, Bruce R. Pitt, Song Li 587 Artificial Viral-Like Gene Delivery System Multifunctional Envelope-type Nano Device Equipped with Transferrin and a pH-Sensitive Fusogenic Peptide Shinji Chaki, Kentaro Sasaki, Kentaro Kogure, Hirofumi Hamada, Masaharu Ueno, Shiroh Futaki, Hideyoshi Harashima 588 Structure-Activity Relationships in Two Series of Pyridinium Gemini Surfactants for Gene Delivery Marc A. Ilies, Betty H. Johnson, William A. Seitz, Ed L. Ezell, Aaron Miller, E. Brad Thompson, Alexandru T. Balaban 589 Construction and Antiproliferative Effect of mc-gðIFN Jiangxue Wu, Bijun Huang, Benling Xu, Gang Xue 590 Efficient, Serum-Resistant Transfection of Murine Squamous Cell Carcinoma Cells by Metafectene and GeneJammer: Application to HSV-tk/Ganciclovir Gene Therapy Krystyna Konopka, Nathan Overlid, Nejat Düzgünes 591 Transfection Efficiency of Monomolecular DNA Nanoparticles Formulated with New Perfluorinated Polycationic Dimerizable Detergents Karine Fabio, Christophe Di Giorgio, Pierre Vierling 592 A Multifunctional Envelope-Type Nano Device for the Novel Gene Delivery of siRNA Plasmids: Optimization of Condensing Polycations Rumiko Moriguchi, Kentaro Kogure, Hidetaka Akita, Siroh Futaki, Makoto Miyagishi, Kazunari Taira, Hideyoshi Harashima 593 Progress towards DNA-Based and siRNA Therapeutics Michael R. Jorgensen, Michael Keller, Eric Perouzel, Andrew D. Miller 594 Enhancement of Cellular and Nuclear Uptake of Macromolecules, Therapeutic Genes and Lipoplexes by Using NLS and PTD Containing Viral Peptides Roosmarijn Vandenbroucke, Niek N. Sanders, Stefaan De Smedt, Joseph Demeester 595 Synthesis of Novel Polyhydroxylalkyleneamine for Nonviral Gene Deliver Li Qun, Guisheng Zhang, Dexi Li 596 Incorporation of a Hepatocyte-Specific Compound in Polycationic Liposomes Enhances Gene Transfection in Hep G2 Cells Michelle R. McCombs, Gideon O. Berger, Michael H. Nantz, Jian Wu Inborn Errors of Metabolism: Hemophilia 597 Liver Tissue Engineering at Extra-Hepatic Site: A Novel Therapeutic Approach toward Hemophilia Kazuo Ohashi, Mark A. Kay, Takashi Yokoyama, Hiroyuki Kuge, Hiromichi Kanehiro, Michiyoshi Hisanaga, Saiho Ko, Hiroyuki Naka, Akira Yoshioka, Yoshiyuki Nakajima 598 In-Vivo Test of Coagulation Factor VIII with Improved Properties for Hemophilia A Gene Therapy by Helper- Dependent Adenoviral Vectors Vincenzo Cerullo, Michael McCormack, Racel Garcia, Christian Clarke, Steven W. Pipe, Brendan Lee 599 Development of EIAV-Based Vectors for the Treatment of Haemophilia A Emily J. Custard, Fraser J. Wilkes, Susan M. Kingsman, Kyriacos A. Mitrophanous, Pippa A. Radcliffe 600 A Novel Gene-Deleted Adenoviral Vector for Somatic Integration and Long-Term Phenotypic Correction of Murine and Canine Hemophilia B Anja Ehrhardt, Hui Xu, Stephen R. Yant, Jeffrey A. Engler, Mark A. Kay 601 Persistent Expression of Factor VIII In Vivo Following FIV Lentiviral Gene Transfer Yubin Kang, Litao Xie, Diane Thi Tran, Colleen S. Stein, Melissa Hickey, Beverly L. Davidson, Paul B. McCray 602 Induction of Tolerance to Human Factor VIII after Neonatal Gene Transfer of a Retroviral Vector Is Dose- Dependent in Mice and Is Not Effective in Cats Lingfei Xu, Manxue Mei, Mark E. Haskins, Karyn Cullen, Katherine P. Ponder
82 8th Annual Meeting • June 1 - 5, 2005 • St. Louis ABSTRACTS FRIDAY, JUNE 3 603 Correction of Hemophilia B Phenotype by Novel Method of Regional Intravenous Delivery of AAV Vector to Skeletal Muscle of Hemophilia B Dogs Valder Arruda, Hansell Stedman, Haiyan Jian, Glenn Pierce, Joerg Schuettrumpf, Christian Furlan Freguia, Stefano Baila, Kapil Gopal, Timothy Nichols, Katherine High 604 Long-Term, Erythroid-Specific Expression of Human Factor IX in Hemophilic Mice Engrafted with Lentiviral Vector-Transduced Hematopoietic Stem Cells Alex H. Chang, Matthias Stephan, Michel Sadelain 605 Efficient AAV 1 and AAV2 Hybrid Vector for Gene Therapy of Hemophilia Bernd Hauck, Ruian Xu, Jing Xie, Qiulan Ding, Wenman Wu, Matthew Sipler, Hongli Wang, Katherine High, Weidong Xiao 606 Molecular Characterization of Hereditary Factor VII Deficiency in Beagles Majed N. Aljamali, Mary Beth Callan, Eleanor S. Pollak, Urs Giger, Petra Werner, Monika Griot-Wenk, Katherine A. High 607 Long-Term Phenotypic Correction of Canine Hemophilia B Following Systemic Administration of Helper Dependent Adenoviral Vector Nicola Brunetti-Pierri, Timothy Nichols, Donna Palmer, Stephanie McCorquodale, Elizabeth Merricks, Arthur Beaudet, Philip Ng 608 Cytotoxic T Lymphocyte Response to AAV2- and AAV8- Mediated Human Factor IX Gene Therapy in Mice Jian Chen, Hui-Chen Hsu, Alex Schlachterman, Katherine A. High, John D. Mountz 609 Long Term Persistence of Canine Factor IX-Specific Th1 Lymphocytes in Regional Lymph Nodes after AAV1- Mediated Intramuscular Gene Transfer in a Hemophilia B Dog Federico Mingozzi, Denise Sabatino, Timothy Nichols, Stephanie McCroquodale, Valder Arruda, Katherine High 610 Evaluation of Three Reticuloendothelial System Blocking Modalities on the Outcome of Adenoviral Mediated Gene Therapy in Hemophilic Mice Maha Othman, Andrea Labelle, Christine Hough, Alan H. Lazarus, David Lillicrap 611 Nonviral Gene Transfer of Factor VII as Alternative Treatment for Hemophilia A Steven C. Chen, Daniel Greenberg, Peiqing Ye, Earl W. Davie, Carol H. Miao 612 Determination of Specific Th1 and Th2 CD4 T Cell Responses in AAV2- and AAV8-Mediated Human Factor IX Gene Therapy in Mice Jian Chen, Hui-Chen Hsu, Alex Schlachterman, Katherine A. High, John D. Mountz 613 Dependence of Vector Dose and Age of Mice in the Induction of Immune Response Against Factor VIII Following Liver-Directed Nonviral Gene Transfer Peiqing Ye, Carol H. Miao 614 Frequency of T Cell Responses to AAV-2 Capsid in Peripheral Blood Mononuclear Cells of Normal Subjects Does Not Correlate with Anti-AAV2 Antibody Titers Denise Sabatino, Federico Mingozzi, Yi-lin Liu, Daniel Hui, Michael Tigges, Glenn Pierce, Katherine High 615 A Self-Limiting but Prolonged Inflammatory Response to AAV-Factor IX Gene Transfer to Skeletal Muscle L. Wang, R. W. Herzog Cardiovascular: Angiogenesis and Vascular Gene Therapy 616 Protection of Monocrotaline-Induced Pulmonary Hypertension by Adeno-Associated Virus Vector- Mediated Interleukin-10 Expression Takayuki Ito, Takashi Okada, Hiroshi Miyashita, Tatsuya Nomoto, Yoshikazu Maeda, Mutsuko Sarukawa, Masahisa Shimpo, Toru Yoshioka, Takashi Matsushita, Hiroaki Mizukami Akihiro Kume, Keiji Yamamoto, Uichi Ikeda, Kazuyuki Shimada, Keiya Ozawa 617 A Synthetic Promoter Drives Macrophage Expression of ApoE and Ameliorates Atherosclerosis Senlin Li, Weijing He, Mei Qiang, Robert A. Reddick, Gregory L. Freeman 618 A Novel Gene Therapy Strategy for Vascular Ehlers- Danlos Syndrome by the Combination with RNAi Mediated Inhibition of a Mutant Allele and Transcriptional Activation of a Normal Allele Atsushi Watanabe, Takahito Wada, Kumiko Tei, Ryuichiro Hata, Yoshimitsu Fukushima, Takashi Shimada
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156 NOTES 8th Annual Meeting • Ju
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FINAL PROGR AM - American Society of Gene & Cell Therapy

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