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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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