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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48<br />
8th Annual Meeting • June 1 - 5, 2005 • St. Louis<br />
ABSTRACTS<br />
THURSDAY, JUNE 2<br />
33 2:30 PM<br />
Non-Random Genomic Distribution <strong>of</strong> Retrovirus<br />
Vector Integration in Successful SCID-X1 <strong>Gene</strong> <strong>Therapy</strong><br />
Kerstin Schwarzwaelder, Manfred Schmidt, Steven Howe,<br />
Hanno Glimm, Claudia Prinz, Manuela Wissler, Annette<br />
Deichmann, Sonja Schmidt, Bobby Gaspar, Adrian Thrasher<br />
Christ<strong>of</strong> von Kalle<br />
34 2:45 PM<br />
Transposon-Based <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> Hemophilia A<br />
Targeting Endothelial <strong>Cell</strong>s in Neonates<br />
Li Liu, Bradley S. Fletcher<br />
35 3:00 PM<br />
<strong>Gene</strong>ralized Detoxification Associated with Engraftment<br />
<strong>of</strong> <strong>Gene</strong>-Corrected Repopulating <strong>Cell</strong>s Achieved in<br />
ADA-SCID Patients by Stem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong> without<br />
Myelopreparative Pre-Conditioning<br />
Makoto Otsu, Satoru Nakajima, Miyuki Kida, Yoshihiro<br />
Maeyama, Nariaki Toita, Norikazu Hatano, Nobuaki<br />
Kawamura, Motohiko Okano, Ryouji Kobayashi, Osamu<br />
Tatsuzawa Masafumi Onodera, Fabio Candotti, Michael S.<br />
Hershfield, Yukio Sakiyama, Tadashi Ariga<br />
36 3:15 PM<br />
Improved Lentiviral Vectors for Systemic <strong>Gene</strong> Transfer<br />
in the Absence <strong>of</strong> an Immune Response<br />
Brian D. Brown, Ehud Hauben, Angelo Lombardo, Lucia<br />
Sergi Sergi, Maria Grazia Roncarolo, Luigi Naldini<br />
37 3:30 PM<br />
A Novel Form <strong>of</strong> Enzyme Replacement <strong>Therapy</strong> for<br />
ADA-Deficiency: In Vivo Transduction by Neonatal<br />
Injection <strong>of</strong> Lentivirus Expressing ADA<br />
Denise A. Carbonaro, Xiangyang Jin, Denise Petersen, Donald<br />
B. Kohn<br />
38 3:45 PM<br />
Inhibition <strong>of</strong> Apolipoprotein B-100 Expression by<br />
Lentivirus Mediated RNA Interference in Mice after<br />
Stem <strong>Cell</strong> Transduction and Transplantation<br />
Mikko P. Turunen, Petri I. Makinen, Pia Leppanen, Juulia<br />
Enback, Suvi Koota, Tero Vatanen, Seppo Yla-Herttuala<br />
39 4:00 PM<br />
Effects <strong>of</strong> Long-Term Expression <strong>of</strong> Activated Murine<br />
FVII in Normal and Hemophilic Mice<br />
Majed N. Aljamali, Paris Margaritis, Rodney M. Camire,<br />
Katherine A. High<br />
40 4:15 PM<br />
Enhanced Factor IX Delivery from Bioengineered Hybrid<br />
Human Skeletal Muscle Co-Expressing VEGF<br />
Lieven Thorrez, Herman Vandenburgh, Desire Collen, Janet<br />
Shansky, Thierry VandenDriessche, Marinee Chuah<br />
Oral Abstract Session 244<br />
Cardiac <strong>Gene</strong> Transfer<br />
Room: 123/127<br />
41 2:00 PM<br />
High Efficiency, Catheter-Based, <strong>Gene</strong> Transfer to the<br />
Large Animal Heart<br />
David M. Kaye, Kenneth Chien, Masahiko Hoshijima,<br />
Krisztina Zsebo, John Power<br />
42 2:15 PM<br />
Uniform Scale-Independent <strong>Gene</strong> Transfer to Striated<br />
Muscle after Transvenular Extravasation <strong>of</strong> Vector<br />
Kapil Gopal, Leonard T. Su, Zhonglin Wang, Xiaoqing Yin,<br />
Anthony Nelson, Benjamin W. Kozyak, James M. Burkman,<br />
Marilyn A. Mitchell, David W. Low, Charles R. Bridges<br />
Hansell H. Stedman<br />
43 2:30 PM<br />
Targeting the Biology <strong>of</strong> Heart Disease: Engineered Zinc<br />
Finger Protein Repressors <strong>of</strong> Phospholamban as a<br />
Potential <strong>Therapy</strong> for Congestive Heart Failure<br />
H. Steve Zhang, Lei Zhang, Yan Huang, Dinggang Liu, Yuxin<br />
Liang, Reed Hickey, Dmitry Guschin, Simon Chandler, Mike<br />
Kunis, Linda Hinh Danny Xia, Xiaohong Zhong, S. Kaye<br />
Spratt, J. Tyler Martin, Casey C. Case, Dale Ando, Edward J.<br />
Rebar, Philip D. Gregory, Frank Giordano<br />
44 2:45 PM<br />
Development <strong>of</strong> AAV-Mediated <strong>Gene</strong> <strong>Therapy</strong> for<br />
Murine Models <strong>of</strong> <strong>Gene</strong>tic Diseases Affecting the Heart<br />
Christina A. Pacak, Cathryn Mah, Gabriel Gaidosh, Melissa<br />
Lewis, Raquel Torres, Kevin Campbell, Glenn A. Walter, Barry<br />
J. Byrne<br />
45 3:00 PM<br />
Adenoviral <strong>Gene</strong> Transfer <strong>of</strong> SERCA2a Restores Mechanical<br />
and Energetic Left-Ventricular Function in<br />
Spontaneously Diabetic Rats<br />
Susumu Sakata, Yuri Sakata, Elie R. Chemaly, Prabhu M.<br />
Padmanabhan, Djamel Lebeche, Miyako Takaki, Federica del<br />
Monte, Roger J. Hajjar<br />
46 3:15 PM<br />
In Vivo Adenoviral <strong>Gene</strong> Transfer <strong>of</strong> Activated PI3-<br />
Kinase Rescues Cardiac Dysfunction and Injury Induced<br />
by Ischemia-Reperfusion after Chronic Cardiac Akt<br />
Activation<br />
Tomohisa Nagoshi, Takashi Matsui, Takuma Aoyama, Ling Li,<br />
David A. Kass, Hunter C. Champion, Anthony Rosenzweig<br />
47 3:30 PM<br />
Ex Vivo Hypothermic Recirculatory Adenoviral <strong>Gene</strong><br />
Transfer to the Transplanted Pig Heart<br />
Keiji Oi, William R. Davies, Henry D. Tazelaar, Kent R.<br />
Bailey, Mark J. Federspiel, Stephen J. Russell, Christopher G.<br />
A. McGregor
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