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FINAL PROGR AM - American Society of Gene & Cell Therapy

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<strong>AM</strong>ERICAN SOCIETY OF GENE THERAPY • Final Program 51<br />

ABSTRACTS<br />

THURSDAY, JUNE 2<br />

78 4:15 PM<br />

Expression <strong>of</strong> the Herpes Simplex Virus Thymidine<br />

Kinase <strong>Gene</strong> by a Promoter Region <strong>of</strong> the Human<br />

SPARC <strong>Gene</strong> Inhibits Human Melanoma <strong>Cell</strong> Growth In<br />

Vitro and In Vivo<br />

Maria V. Lopez, Patricia V. Blanco, Diego L. Viale, Eduardo A.<br />

Cafferata, David Gould, Yuti Chernajovsky, Osvaldo Podhajcer<br />

Oral Abstract Session 248<br />

<strong>Gene</strong> Regulation: Keeping <strong>Gene</strong>s On and Turning<br />

Them Off<br />

Room: 261/262<br />

79 2:00 PM<br />

Real Time Fluorescent Tracking <strong>of</strong> Dynamic Variegation<br />

by Retrovirus Vectors in Stem <strong>Cell</strong>s<br />

James Ellis, John Ramunas, Liam Kelly, Tanya Sukonnik, Eric<br />

Jervis<br />

80 2:15 PM<br />

Insulation <strong>of</strong> Oncoretrovirus Vectors with the cHS4<br />

Chromatin Insulator Is Associated with an Open Chromatin<br />

Histone Code<br />

Changlong Li, David W. Emery<br />

81 2:30 PM<br />

A Retrovirus Vector Screen for the Identification <strong>of</strong><br />

Novel Chromatin Insulators and Enhancers<br />

Amy C. Groth, George Stamatoyannopoulos, David W. Emery<br />

82 2:45 PM<br />

Targeted Pancreatic Cancer-Specific <strong>Gene</strong> <strong>Therapy</strong><br />

Controlled by a Molecular-Engineered Human Cholecystokinin<br />

Type-A Receptor (CCKAR) Promoter in In<br />

Vivo Imaging Models<br />

Xiaoming Xie, Zheng Li, Qingqing Ding, Hus-Ping Kuo,<br />

James L. Abbruzzese, Mien-Chie Hung<br />

83 3:00 PM<br />

Persistent and Position Independent Transgene Expression<br />

in Erythroid <strong>Cell</strong>s Transduced by Lentiviral Vectors<br />

Annarita Miccio, Francesco Lotti, Claudia Rossi, Giuliana<br />

Ferrari<br />

84 3:15 PM<br />

Assessing Hemostatic Efficacy <strong>of</strong> Continuous Expression<br />

<strong>of</strong> Factor VIIa after AAV-Mediated, Liver-Directed <strong>Gene</strong><br />

Transfer in Hemophilia A and B Mice<br />

Alexander Schlachterman, Paris Margaritis, Majed Aljamali,<br />

Jianhua Liu, Valder Arruda, Katherine High<br />

85 3:30 PM<br />

Development <strong>of</strong> Zinc Finger Nucleases for Therapeutic<br />

<strong>Gene</strong> Correction <strong>of</strong> Sickle <strong>Cell</strong> Anemia<br />

Jeffrey C. Miller, Fyodor D. Urnov, Ya-li Lee, Jeremy Rock,<br />

Tyler Sorba, Sarah Patterson, Philip D. Gregory, Michael C.<br />

Holmes, Edward J. Rebar<br />

86 3:45 PM<br />

Transcriptional <strong>Gene</strong> Silencing in Human <strong>Cell</strong>s<br />

Zhaoxia Chen, Ali Ehsani, Riggs Art, Rossi J. John, Kevin V.<br />

Morris<br />

87 4:00 PM<br />

Determinants <strong>of</strong> Toxicity Induced by In Vivo Expression<br />

<strong>of</strong> Short Hairpin RNA from AAV Vectors<br />

Dirk Grimm, Konrad L. Streetz, Kusum Pandey, Theresa A.<br />

Storm, Mark A. Kay<br />

88 4:15 PM<br />

Driving shRNA Transcription from PolII Promoters in<br />

Mammalian <strong>Cell</strong>s<br />

Jeffery C. Giering, Mark A. Kay<br />

Poster Session I<br />

Exhibit Hall 2<br />

RNA Virus Vectors: <strong>Gene</strong> Expression<br />

89<br />

Amelioration <strong>of</strong> Gamma Retroviral Silencing during<br />

Embryonic Stem <strong>Cell</strong>-Derived Hematopoiesis: Preclinical<br />

Assessment <strong>of</strong> Safety-Improved Vectors<br />

Ali Ramezani, Teresa S. Hawley, Robert G. Hawley<br />

90<br />

In Vitro and In Vivo <strong>Gene</strong> Expression <strong>of</strong> Lentiviral<br />

Vectors in CD4+ T <strong>Cell</strong>s<br />

Kit L. Shaw, Karen Pepper, Denise Petersen, Vesa Kaartinen,<br />

Donald B. Kohn<br />

91<br />

Insertion <strong>of</strong> the cHS4-Insulator into Lentiviral Vectors:<br />

Use <strong>of</strong> Real-Time PCR in Order To Analyze the Effects<br />

on Transgene Expression and Viral Titre<br />

Johan Jakobsson, Troels Tolstrup Nielsen, Nina Rosenqvist,<br />

Cecilia Lundberg<br />

92<br />

Efficient Trangene Expression in Central Nervous System<br />

through a Non-Integrative Lentiviral Vector<br />

Stephanie Philippe, Che Serguera, Sebastien Bonnel, Christelle<br />

Vetu, Martine Barkats, Caroline Petit, Marc Abitbol, Chamsy<br />

Sarkis, Jacques Mallet<br />

93<br />

Diminished Mobilization <strong>of</strong> Self-Inactivating (SIN)<br />

Lentiviral Vectors Containing Globin Regulatory Elements<br />

Compared to Those Containing a Retroviral Long<br />

Terminal Repeat<br />

Hideki Hanawa, Derek A. Persons, Takashi Shimada, Arthur<br />

W. Nienhuis<br />

94<br />

Transgene Expression with Integration-Deficient HIV-1<br />

Based Lentiviral Vectors Expressing Class I Integrase<br />

Mutants<br />

G. Joseph, J. Marsh, T. Johnson, K. Cornetta

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