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RAC March 15, 2005 - Presentation Materials
99th Meeting
Bethesda, Maryland

Welcome  
  Amy P. Patterson, M.D., Office of Biotechnology Activities, NIH
Diane Wara, M.D., Chair, NIH RAC
 
  Slides  Webcast   
 

Keynote Speaker  
  Theodore Friedmann, M.D., University of California, San Diego  
  Slides  Webcast   
 

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
 
  Webcast   
 

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  
  Webcast   
 

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  
  Slides  Webcast   
 

Retroviral Gene Transfer for Treatment of X-SCID and ADA-SCID  
  Fabio Candotti, M.D., National Human Genome Research Institute, NIH  
  Slides  Webcast   
 

Summary of Recent Safety Data Emerging from French X-SCID Trial  
  Theodore Friedmann, M.D., University of California, San Diego  
  Webcast   
 

U.S. Food and Drug Administration Perspectives  
  Carolyn A. Wilson, Ph.D., U.S. Food and Drug Administration  
  Slides  Webcast   
 

Discussion  
  Webcast   
 

Retrovirus Integration and Insertional Mutagenesis  
  Moderators:
Naomi Rosenberg, Ph.D., Tufts University
Marina O�Reilly, Ph.D., Office of Biotechnology Activities, NIH
 
  Webcast   
  Gene Transfer and Insertional Mutagenesis  
  Christof von Kalle, M.D., Cincinnati Children�s Hospital Medical Center  
  Slides
 
  Gene Transfer Insertional Mutagenesis Insights  
  Neal G. Copeland, Ph.D., National Cancer Institute, NIH  
  Slides
 
  Retroviral Integration Target Site Preferences  
  Frederic D. Bushman, Ph.D., University of Pennsylvania  
  Slides
 
  Discussion  
  Presentation Questions   
 

Bone Marrow Transplantation for SCID  
  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
 
  Webcast   
  Overview of HLA Identical and Haploidentical BMT: Duke Experience  
  Rebecca H. Buckley, M.D., Duke University Medical Center  
  Slides
 
  Overview of HLAIdentical and Haploidentical BMT: Memorial Sloan-Kettering Experience  
  Richard J. O�Reilly, M.D., Memorial Sloan-Kettering Cancer Center  
  Slides
 
  Overview of Cord Blood Transplantation  
  John E. Wagner, M.D., University of Minnesota  
  Slides
 

Discussion / Wrap-Up and Next Steps  
  Moderators:
Theodore Friedmann, M.D., University of California, San Diego
Amy P. Patterson, M.D., Office of Biotechnology Activities, NIH
Diane Wara, M.D., Chair, NIH RAC
 
  Webcast   
 

ADJOURN  
 

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