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Welcome |
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Amy P. Patterson, M.D., Office of Biotechnology Activities, NIH
Diane Wara, M.D., Chair, NIH RAC |
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Keynote Speaker |
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Theodore Friedmann, M.D., University of California, San Diego |
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Slides
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Current SCID Gene Transfer Experience |
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Moderators:
Diane W. Wara, M.D., University of California, San Francisco
Bernard Lo, M.D., University of California, San Francisco |
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Approaches to the Patient with X-Linked SCID Who Has Failed Standard BMT Treatment |
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Jennifer M. Puck, M.D., National Human Genome Research Institute, NIH |
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Preliminary Results of an NIH Trial of Retroviral IL2RG Gene Transfer as Salvage Treatment for Older Post-BMT SCID Patients |
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Harry L. Malech, M.D., National Institute of Allergy and Infectious Diseases, NIH |
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Slides
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Retroviral Gene Transfer for Treatment of X-SCID and ADA-SCID |
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Fabio Candotti, M.D., National Human Genome Research Institute, NIH |
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Slides
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Summary of Recent Safety Data Emerging from French X-SCID Trial |
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Theodore Friedmann, M.D., University of California, San Diego |
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U.S. Food and Drug Administration Perspectives |
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Carolyn A. Wilson, Ph.D., U.S. Food and Drug Administration |
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Slides
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Discussion |
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Retrovirus Integration and Insertional Mutagenesis |
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Moderators:
Naomi Rosenberg, Ph.D., Tufts University
Marina O'Reilly, Ph.D., Office of Biotechnology Activities, NIH |
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Gene Transfer and Insertional Mutagenesis |
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Christof von Kalle, M.D., Cincinnati Children's Hospital Medical Center |
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Slides
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Gene Transfer Insertional Mutagenesis Insights |
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Neal G. Copeland, Ph.D., National Cancer Institute, NIH |
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Slides
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Retroviral Integration Target Site Preferences |
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Frederic D. Bushman, Ph.D., University of Pennsylvania |
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Slides
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Discussion |
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Presentation Questions
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Bone Marrow Transplantation for SCID |
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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 |
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Overview of HLA Identical and Haploidentical BMT: Duke Experience |
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Rebecca H. Buckley, M.D., Duke University Medical Center |
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Slides
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Overview of HLAIdentical and Haploidentical BMT: Memorial Sloan-Kettering Experience |
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Richard J. O'Reilly, M.D., Memorial Sloan-Kettering Cancer Center |
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Slides
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Overview of Cord Blood Transplantation |
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John E. Wagner, M.D., University of Minnesota |
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Slides
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Discussion / Wrap-Up and Next Steps |
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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 |
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ADJOURN |
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