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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60<br />
8th Annual Meeting • June 1 - 5, 2005 • St. Louis<br />
ABSTRACTS<br />
THURSDAY, JUNE 2<br />
233<br />
Towards Reversing Sandh<strong>of</strong>f Pathology by Lentiviral-<br />
Mediated <strong>Gene</strong> <strong>Therapy</strong><br />
Audrey Arfi, Rivka Ziesling, Livia Poenaru, Anthony H.<br />
Futerman, Catherine Caillaud<br />
234<br />
Polyclonal Insertion Sites in Two Rhesus Macaques after<br />
Non-Myeloablative Transplantation with MFGS-gp91phox Transduced Autologous CD34+PBPC<br />
Sebastian Brenner, Martin F. Ryser, Uimook Choi, Narda<br />
Whiting-Theobald, Gilda Linton, Elizabeth Kang, Romy<br />
Lehmann, Anne M. Farese, Thomas J. MacVittie, Joachim<br />
Roesler Mitchell E. Horwitz, Harry L. Malech<br />
235<br />
The Route <strong>of</strong> Vector Delivery Impacts the Therapeutic<br />
Effects <strong>of</strong> AAV-Mediated <strong>Gene</strong> <strong>Therapy</strong> in Adult<br />
Mucopolysaccharidosis IIIB Mice<br />
Haiyan Fu, Lu Kang, Sheryl S. Moy, Jerillyn S. Jennings,<br />
Antonio Perez, Joseph Muenzer<br />
236<br />
Bone Marrow-Derived <strong>Cell</strong>s Do Not Contribute to<br />
Endocrine Pancreas Regeneration in Diabetic RIP/IGF-I<br />
Transgenic Mice<br />
Eduard Ayuso, Veronica Jimenez, Judith Agudo, Virginia<br />
Haurigot, Jose Carlos Segovia, Juan Bueren, Fatima Bosch<br />
237<br />
Evaluation <strong>of</strong> Low-Level, Constitutive Expression <strong>of</strong> bðglucuronidase<br />
on the Clinical Manifestations <strong>of</strong><br />
Mucopolysaccharidosis Type VII<br />
Anthony Donsante, Beth Levy, Carole Vogler, Mark Sands<br />
238<br />
Stem <strong>Cell</strong> <strong>Gene</strong> Delivery to Immunocompetent Mice<br />
Using a Non-Myeloablative Regimen Results in Long-<br />
Term Transgene Expression and Stable Mixed Chimerism<br />
Brian W. Bigger, Simon N. Waddington, Mamta Buch, Megha<br />
Nivsarkar, Nicholas Jones, Suzanne M. Watt, Kathryn Wood,<br />
Michael Themis<br />
239<br />
Immunomodulation Is Necessary to Achieve Stable<br />
Expression after Retroviral Vector-Mediated Liver-<br />
Directed <strong>Gene</strong> <strong>Therapy</strong> to Adult Mice with MPS I<br />
Xiucui Ma, Lingfei Xu, Yuli Liu, Bin Wang, Katherine P. Ponder<br />
240<br />
Intramuscular Administration <strong>of</strong> an AAV Vector Expressing<br />
4-Sulfatase (4S) to Newborn Cats With<br />
Mucopolysaccaridosis VI: Sulfatase Activity in Muscle,<br />
Serum, and Liver<br />
Mark Haskins, Thomas O’Malley, Ping Wang, Karyn Cullen,<br />
Anne Traas, Alessandra Tessitore, Monica Doria, Alberto<br />
Auricchio<br />
241<br />
<strong>Gene</strong> Expression in the Striatum <strong>of</strong> HPRT Knockout<br />
Mice<br />
Song Shaochun, Theodore Friedmann<br />
242<br />
The Use <strong>of</strong> AAV Serotype 1 Vector for Expression <strong>of</strong> að-<br />
Galactosidase A in Fabry Knockout Mice<br />
Koichi Ogawa, Yukihiko Hirai, Katsuko Zenri-Aihara,<br />
Yoshitaka Fukunaga, Takashi Shimada<br />
243<br />
Transplantation <strong>of</strong> Neonatal NOD/SCID/MPSVII Mice<br />
with Human Mesenchymal Stem <strong>Cell</strong>s<br />
Phillip E. Herrbrich, Todd E. Meyerrose, Alex A. H<strong>of</strong>ling,<br />
Mark Sands, Jan A. Nolta<br />
244<br />
AAV Vector-Mediated <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong><br />
Mucopolysaccharidosis Type I<br />
Lalitha Belur, Damaris Lorenzo, Elena Aronovich, Walter C.<br />
Low, Chester B. Whitley, R. Scott McIvor<br />
245<br />
AAV2/8-Mediated Efficacy and Immune Tolerance in a<br />
Pompe Disease Mouse Model<br />
S. D. Bercury, R. J. Ziegler, C. M. Barbon, D. Souza, D.<br />
Armentano, B. L. Thurberg, T. Taksir, H. Do, M. Trout, N.<br />
Raben S. H. Cheng<br />
246<br />
Comparison <strong>of</strong> rAAV2-Mediated Expression <strong>of</strong> Human<br />
and Murine β-Glucuronidase in Adult MPS VII Mice<br />
Kristin C. Greathouse, Chiou-Miin Wang, Thomas J. Sferra<br />
247<br />
<strong>Gene</strong> <strong>Therapy</strong> with a Retroviral Vector Expressing<br />
Canine β-Glucuronidase to Juvenile (7 Week Old ) Dogs<br />
Improves the Biochemical Manifestations <strong>of</strong> MPS VII<br />
Bin Wang, Ping Wang, Tom O’Malley, Lingfei Xu, Mark E.<br />
Haskins, Katherine P. Ponder<br />
248<br />
A Mouse Model <strong>of</strong> Type VII Glycogenosis to Assay <strong>Gene</strong><br />
<strong>Therapy</strong> Approaches<br />
Fatima Bosch, Miquel Garcia, Anna Pujol, Albert Ruzo, Anna<br />
Arbos, Juan E. Feliu<br />
249<br />
Minimal Dose <strong>of</strong> AAV8/DC190-ASM Necessary for<br />
Therapeutic Efficacy in a Mouse Model <strong>of</strong> Niemann-<br />
Pick Disease<br />
R. J. Ziegler, C. M. Barbon, S. D. Bercury, M. L. Hawes, E.<br />
H. Schuchman, R. J. Desnick, S. H. Cheng<br />
250<br />
Correction <strong>of</strong> Hunter Syndrome in the Ids-Knock-Out<br />
Mouse Model by r-AVV Mediated <strong>Gene</strong> <strong>Therapy</strong><br />
Monica Cardone, Vinicia Polito, Alessandra D’Azzo, Joseph<br />
Muenzer, Alberto Auricchio, Andrea Ballabio, Maria Pia Cosma
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