National Institutes of Health
Recombinant DNA Advisory Committee (RAC)
Gene Transfer Safety Symposium:
Current Perspectives on Gene Transfer for X-SCID
Bethesda Marriott Hotel
Bethesda , Maryland
Tuesday, March 15, 2005
CAPTION TEXT (HTML)
NOTE: The caption text is a record of the close captioning offered to support the video. It is not a totally accurate, verbatim transcript of the meeting. It is offered here for interest and support of the video. A correct transcript will be available on the NIH web site in due course.
Symposium Agenda
8:00 AM Welcome (Real Video)
- Amy P. Patterson, M.D., Office of Biotechnology Activities, NIH
- Diane W. Wara, M.D., University of California, San Francisco
Presentation (PDF 3MB)
8:10 AM Keynote Speaker
Theodore Friedmann, M.D., University of California, San Diego
Presentation (PDF 439k)
(Real Video)
8:25 AM Current SCID Gene Transfer Experience
Moderators: Diane W. Wara, M.D., University of California, San Francisco
Bernard Lo, M.D., University of California, San Francisco
(Real Video)
Approaches to the Patient with X-Linked SCID Who Has Failed Standard BMT Treatment
Jennifer M. Puck, M.D., National Human Genome Research Institute, NIH
(Real Video)
Preliminary Results of an NIH Trial of Retroviral IL2RG Gene Transfer as Salvage Treatment for Older Post-BMT SCID Patients
Harry L. Malech, M.D., National Institute of Allergy and Infectious Diseases, NIH
Presentation (PDF 1.6MB)
(Real Video)
Retroviral Gene Transfer for Treatment of X-SCID and ADA-SCID
Fabio Candotti, M.D., National Human Genome Research Institute, NIH
Presentation (PDF 692k)
(Real Video)
Summary of Recent Safety Data Emerging from French X-SCID Trial
Theodore Friedmann, M.D., University of California, San Diego
(Real Video)
U.S. Food and Drug Administration Perspectives
Carolyn A. Wilson, Ph.D., U.S. Food and Drug Administration
Presentation (PDF 53k)
(Real Video)
10:15 AM Discussion (Real Video)
10:30 AM - BREAK
10:45 AM Cont Discussion (Real Video)
11:30 AM Retrovirus Integration and Insertional Mutagenesis
(Real Video)
Moderators: Naomi Rosenberg, Ph.D., Tufts University
Marina O’Reilly, Ph.D., Office of Biotechnology Activities, NIH
Gene Transfer and Insertional Mutagenesis
Christof von Kalle, M.D., Cincinnati Children’s Hospital Medical Center
Presentation (PDF 2.2MB)
Gene Transfer Insertional Mutagenesis Insights
Neal G. Copeland, Ph.D., National Cancer Institute, NIH
Presentation (PDF 1.6MB)
Retroviral Integration Target Site Preferences
Frederic D. Bushman, Ph.D., University of Pennsylvania
Presentation (PDF 1MB)
12:45 PM Discussion
Disscusion Questions Presentation (PDF 1.3MB)
1:30 PM Lunch
2:30 PM Bone Marrow Transplantation for SCID
(Real Video)
Moderators: Bernard Lo, M.D., University of California, San Francisco
- Theodore Friedmann, M.D., University of California, San Diego
- Thomas Holohan, M.D., Office of Biotechnology Activities, NIH
Overview of HLA Identical and Haploidentical BMT: Duke Experience
Rebecca H. Buckley, M.D., Duke University Medical Center
Presentation (PDF 675k)
Overview of HLAIdentical and Haploidentical BMT: Memorial Sloan-Kettering Experience Richard J. O’Reilly, M.D., Memorial Sloan-Kettering Cancer Center
Presentation (PDF 868k)
Overview of Cord Blood Transplantation
John E. Wagner, M.D., University of Minnesota
Presentation (PDF 921k)
4:00 PM - BREAK
4:15 PM - Discussion / Wrap-Up and Next Steps
(Real Video)
Moderators: Theodore Friedmann, M.D., University of California, San Diego
- Diane W. Wara, M.D., University of California, San Francisco
- Amy P. Patterson, M.D., Office of Biotechnology Activities, NIH
6:00 PM Adjournment
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