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Office of Biotechnology Activities
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National Institutes of Health - Office of Science Policy
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| Current Perspectives on Gene Transfer for X-linked Severe Combined Immunodeficiency Disease (X-SCID) |
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On December 4-5, 2002, February 10, 2003, March 15, 2005, March 14, 2007 and March 11, 2008, the RAC reviewed the clinical and molecular data concerning three adverse events that occurred in a French study involving engraftment of a CD34+ hematopoietic stem cell enriched, cell population transduced with a retroviral vector encoding the common gamma chain (γ) transmembrane protein subunit shared by receptors for Interleukins 2, 4, 7, 9, 15 and 21. The leukemias appear to share the common causative mechanism of insertional mutagenesis at or near oncogenes. The major goal of the symposia was to increase awareness in the scientific community and the public by providing comprehensive updates of:
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The current US and international trials using gene transfer for SCID, including recently emerging safety data |
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Retrovirus integration and insertional mutagenesis research |
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The use of bone marrow/stem cell transplantation as treatment for SCID |
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The RAC developed recommendations for the use of gene transfer for X-SCID and other indications using retroviral vectors.
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March 11, 2008 |
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Agenda and Slides |
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Webcast |
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March 14, 2007 |
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Agenda |
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Webcasts and Minutes |
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Slides |
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March 15, 2005 |
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Agenda |
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Slides |
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Summary |
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References |
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Conclusions and Recommendations of the RAC Gene Transfer Safety Symposium: Current Perspectives on Gene Transfer for X-SCID (March 15, 2005) |
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February 10, 2003 |
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Webcast
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Conclusions and Recommendations of the RAC Regarding Adverse Events in a Gene Transfer Trial Studying X-linked SCID (March 20, 2003) |
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December 4-5, 2002 |
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Minutes |
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Slides- December 4, 2002 |
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Slides - December 5, 2002 |
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