FINAL PROGR AM - American Society of Gene & Cell Therapy
FINAL PROGR AM - American Society of Gene & Cell Therapy
FINAL PROGR AM - American Society of Gene & Cell Therapy
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82<br />
8th Annual Meeting • June 1 - 5, 2005 • St. Louis<br />
ABSTRACTS<br />
FRIDAY, JUNE 3<br />
603<br />
Correction <strong>of</strong> Hemophilia B Phenotype by Novel<br />
Method <strong>of</strong> Regional Intravenous Delivery <strong>of</strong> AAV Vector<br />
to Skeletal Muscle <strong>of</strong> Hemophilia B Dogs<br />
Valder Arruda, Hansell Stedman, Haiyan Jian, Glenn Pierce,<br />
Joerg Schuettrumpf, Christian Furlan Freguia, Stefano Baila,<br />
Kapil Gopal, Timothy Nichols, Katherine High<br />
604<br />
Long-Term, Erythroid-Specific Expression <strong>of</strong> Human<br />
Factor IX in Hemophilic Mice Engrafted with Lentiviral<br />
Vector-Transduced Hematopoietic Stem <strong>Cell</strong>s<br />
Alex H. Chang, Matthias Stephan, Michel Sadelain<br />
605<br />
Efficient AAV 1 and AAV2 Hybrid Vector for <strong>Gene</strong><br />
<strong>Therapy</strong> <strong>of</strong> Hemophilia<br />
Bernd Hauck, Ruian Xu, Jing Xie, Qiulan Ding, Wenman Wu,<br />
Matthew Sipler, Hongli Wang, Katherine High, Weidong Xiao<br />
606<br />
Molecular Characterization <strong>of</strong> Hereditary Factor VII<br />
Deficiency in Beagles<br />
Majed N. Aljamali, Mary Beth Callan, Eleanor S. Pollak, Urs<br />
Giger, Petra Werner, Monika Griot-Wenk, Katherine A. High<br />
607<br />
Long-Term Phenotypic Correction <strong>of</strong> Canine Hemophilia<br />
B Following Systemic Administration <strong>of</strong> Helper<br />
Dependent Adenoviral Vector<br />
Nicola Brunetti-Pierri, Timothy Nichols, Donna Palmer,<br />
Stephanie McCorquodale, Elizabeth Merricks, Arthur Beaudet,<br />
Philip Ng<br />
608<br />
Cytotoxic T Lymphocyte Response to AAV2- and AAV8-<br />
Mediated Human Factor IX <strong>Gene</strong> <strong>Therapy</strong> in Mice<br />
Jian Chen, Hui-Chen Hsu, Alex Schlachterman, Katherine A.<br />
High, John D. Mountz<br />
609<br />
Long Term Persistence <strong>of</strong> Canine Factor IX-Specific Th1<br />
Lymphocytes in Regional Lymph Nodes after AAV1-<br />
Mediated Intramuscular <strong>Gene</strong> Transfer in a Hemophilia<br />
B Dog<br />
Federico Mingozzi, Denise Sabatino, Timothy Nichols,<br />
Stephanie McCroquodale, Valder Arruda, Katherine High<br />
610<br />
Evaluation <strong>of</strong> Three Reticuloendothelial System Blocking<br />
Modalities on the Outcome <strong>of</strong> Adenoviral Mediated<br />
<strong>Gene</strong> <strong>Therapy</strong> in Hemophilic Mice<br />
Maha Othman, Andrea Labelle, Christine Hough, Alan H.<br />
Lazarus, David Lillicrap<br />
611<br />
Nonviral <strong>Gene</strong> Transfer <strong>of</strong> Factor VII as Alternative<br />
Treatment for Hemophilia A<br />
Steven C. Chen, Daniel Greenberg, Peiqing Ye, Earl W. Davie,<br />
Carol H. Miao<br />
612<br />
Determination <strong>of</strong> Specific Th1 and Th2 CD4 T <strong>Cell</strong><br />
Responses in AAV2- and AAV8-Mediated Human Factor<br />
IX <strong>Gene</strong> <strong>Therapy</strong> in Mice<br />
Jian Chen, Hui-Chen Hsu, Alex Schlachterman, Katherine A.<br />
High, John D. Mountz<br />
613<br />
Dependence <strong>of</strong> Vector Dose and Age <strong>of</strong> Mice in the<br />
Induction <strong>of</strong> Immune Response Against Factor VIII<br />
Following Liver-Directed Nonviral <strong>Gene</strong> Transfer<br />
Peiqing Ye, Carol H. Miao<br />
614<br />
Frequency <strong>of</strong> T <strong>Cell</strong> Responses to AAV-2 Capsid in<br />
Peripheral Blood Mononuclear <strong>Cell</strong>s <strong>of</strong> Normal Subjects<br />
Does Not Correlate with Anti-AAV2 Antibody Titers<br />
Denise Sabatino, Federico Mingozzi, Yi-lin Liu, Daniel Hui,<br />
Michael Tigges, Glenn Pierce, Katherine High<br />
615<br />
A Self-Limiting but Prolonged Inflammatory Response to<br />
AAV-Factor IX <strong>Gene</strong> Transfer to Skeletal Muscle<br />
L. Wang, R. W. Herzog<br />
Cardiovascular: Angiogenesis and Vascular <strong>Gene</strong><br />
<strong>Therapy</strong><br />
616<br />
Protection <strong>of</strong> Monocrotaline-Induced Pulmonary<br />
Hypertension by Adeno-Associated Virus Vector-<br />
Mediated Interleukin-10 Expression<br />
Takayuki Ito, Takashi Okada, Hiroshi Miyashita, Tatsuya<br />
Nomoto, Yoshikazu Maeda, Mutsuko Sarukawa, Masahisa<br />
Shimpo, Toru Yoshioka, Takashi Matsushita, Hiroaki<br />
Mizukami Akihiro Kume, Keiji Yamamoto, Uichi Ikeda,<br />
Kazuyuki Shimada, Keiya Ozawa<br />
617<br />
A Synthetic Promoter Drives Macrophage Expression <strong>of</strong><br />
ApoE and Ameliorates Atherosclerosis<br />
Senlin Li, Weijing He, Mei Qiang, Robert A. Reddick, Gregory<br />
L. Freeman<br />
618<br />
A Novel <strong>Gene</strong> <strong>Therapy</strong> Strategy for Vascular Ehlers-<br />
Danlos Syndrome by the Combination with RNAi<br />
Mediated Inhibition <strong>of</strong> a Mutant Allele and Transcriptional<br />
Activation <strong>of</strong> a Normal Allele<br />
Atsushi Watanabe, Takahito Wada, Kumiko Tei, Ryuichiro<br />
Hata, Yoshimitsu Fukushima, Takashi Shimada
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