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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66<br />
8th Annual Meeting • June 1 - 5, 2005 • St. Louis<br />
ABSTRACTS<br />
THURSDAY, JUNE 2<br />
335<br />
Correction <strong>of</strong> a Murine Model <strong>of</strong> Von Willebrand<br />
Disease by <strong>Gene</strong> Transfer<br />
Robert G. Pergolizzi, Jin Guang, Diane Chan, Denisa Wagner,<br />
Peter Lenting, Ronald G. Crystal<br />
336<br />
Correction <strong>of</strong> Chronic Granulomatous Disease by <strong>Gene</strong><br />
<strong>Therapy</strong><br />
Manuel Grez, Marion G. Ott, Stefan Stein, Ulrich Siler,<br />
Ulrike Koehl, Hana Kunkel, Andrea Schilz, Klaus Kuehlcke,<br />
Dieter Hoelzer, Reinhard Seger<br />
337<br />
Towards <strong>Gene</strong> Correction <strong>Therapy</strong> for Wiskott-Aldrich<br />
Syndrome with Engineered Zinc Finger Nucleases<br />
Lei Zhang, Fyodor D. Urnov, Jeffrey C. Miller, Christian<br />
Beausejour, Ya-Li Lee, Jeremy Rock, Kenneth Kim, Michael C.<br />
Holmes, Philip D. Gregory<br />
338<br />
Non-Viral <strong>Gene</strong> Transfer into Hematopoietic <strong>Cell</strong>s <strong>of</strong><br />
Artemis Deficient ScidA Mice Using the Sleeping Beauty<br />
Transposon System<br />
Andy Wilber, Shannon Buckley, Uma Lakshmipathy, Joel<br />
Frandsen, Morton J. Cowan, R. Scott McIvor<br />
339<br />
Long-Term Protein Transduction Domain Mediated<br />
Intra-<strong>Cell</strong>ular Delivery <strong>of</strong> Purine Nucleoside Phosphorylase<br />
(PNP) Corrects PNP Deficiency in Mice<br />
Ana Toro, Eyal Grunebaum<br />
340<br />
Drug-Selectable as Well as Therapeutic <strong>Gene</strong> Transfer<br />
into Hematopoietic Stem <strong>Cell</strong>s May Be Essential in <strong>Gene</strong><br />
<strong>Therapy</strong> for Inherited Bleeding Disorders <strong>of</strong> Large<br />
Animals<br />
David A. Wilcox, Juan Fang, Lily M. Du, Mary K. Boudreaux<br />
341<br />
Lentiviral Vector-Mediated <strong>Gene</strong> <strong>Therapy</strong> as Treatment<br />
for Wiskott-Aldrich Syndrome (WAS): Pre-Clinical<br />
Studies in Human <strong>Cell</strong> Lines and WASp -/- Mice<br />
S. Humblet-Baron, S. Anover, K. Kipp, Q. Zhu, P. Ye, W.<br />
Zhang, Y. Ovechkina, S. Khim, A. Astrakhan, T. Strom D.<br />
Kohn, F. Candotti, Y. Vyas, H. Ochs, C. Miao, D. Rawlings<br />
342<br />
Human Erythropoietin <strong>Gene</strong> <strong>Therapy</strong> for Patients with<br />
Chronic Renal Failure<br />
Yitzhak Lippin, Michal Dranitzki-Elhale, Einat Brill-Almon,<br />
Chava Mei–Zahav, Sarah Mizrachi, Yael Liberman, Adrian<br />
Iaina, Eli Kaplan, Eduardo Podjarny, Nicole Casadevall Noam<br />
Shani, Eithan Galun<br />
343<br />
Repair <strong>of</strong> Thalassemic bð-Globin pre-mRNA by<br />
Antisense AAV and Lentiviral Vectors in <strong>Cell</strong> Culture<br />
Thipparat Suwanmanee, Vivian W. Choi, Richard J. Samulski,<br />
Tal Kafri, Ryszard Kole<br />
344<br />
Application <strong>of</strong> a suicide <strong>Gene</strong> to X-SCID <strong>Gene</strong> <strong>Therapy</strong><br />
Toru Uchiyama, Satoru Kumaki, Masahumi Onodera, Du Wei,<br />
Looi Chung Yeng, Yoichi Sasahara, Sigeru Tsuchiya<br />
345<br />
<strong>Gene</strong> <strong>Therapy</strong> for Wiskott-Aldrich Syndrome Using<br />
Lentiviral Vectors: Evidence for Efficacy and Safety after<br />
Transduction <strong>of</strong> Human T <strong>Cell</strong>s and Hematopoietic Stem<br />
<strong>Cell</strong>s<br />
Loïc Dupré, Samantha Scaramuzza, Francesco Marangoni,<br />
Sara Trifari, Silvana Martino, Shigeru Tsuchiya, Adrian<br />
Thrasher, Anne Galy, Luigi Naldini, Alessandro Aiuti Maria<br />
Grazia Roncarolo<br />
346<br />
<strong>Gene</strong> Correction <strong>of</strong> X-Linked SCID Using Engineered<br />
Zinc Finger Nucleases and Integration Defective<br />
Lentiviral Delivery<br />
Angelo Lombardo, Christian Beausejour, Fyodor D. Urnov,<br />
Jeffrey C. Miller, Michael C. Holmes, Philip D. Gregory, Luigi<br />
Naldini<br />
347<br />
Long-Term <strong>Gene</strong> Expression and Phenotypic Correction<br />
<strong>of</strong> FANCC Deficient Lymphoblastoid <strong>Cell</strong>s Using the<br />
Sleeping Beauty Transposon System<br />
Shannon A. Wadman, Karl J. Clark, Perry B. Hackett, R Scott<br />
McIvor, Jeffrey J. Essner<br />
348<br />
Integration Site Analysis <strong>of</strong> a Helper-Dependent Adenovirus<br />
Vector Containing AAV ITRs and 22 kb <strong>of</strong> the<br />
Human Globin LCR<br />
Hongjie Wang, Tobias Leege, Dmitry Shayakhmetov, Qiliang<br />
Li, Thalia Papayannopoulou, George Stamatoyannopoulos,<br />
Andre Lieber<br />
349<br />
Partial Correction <strong>of</strong> IL-12 Receptor beta-1 (IL-12Rb1)<br />
Deficiency in Mice upon Transplantation <strong>of</strong> Retrovirally<br />
Transduced Hematopoietic Stem <strong>Cell</strong>s<br />
Marita Bosticardo, Charles Scanga, Francesco Novelli, Jean-<br />
Laurent Casanova, Fabio Candotti<br />
<strong>Gene</strong> <strong>Therapy</strong> Approaches to Pulmonary Disease<br />
350<br />
Pre-Clinical Assays for Cystic Fibrosis <strong>Gene</strong> <strong>Therapy</strong><br />
Uta Griesenbach, on behalf <strong>of</strong> the UK Cystic Fibrosis <strong>Gene</strong><br />
<strong>Therapy</strong> Consortium
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