News FocusHEMOPHILIA

After a Setback, Gene Therapy Progresses ... Gingerly

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Science  02 Mar 2001:
Vol. 291, Issue 5509, pp. 1692-1697
DOI: 10.1126/science.291.5509.1692

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Summary

Amid all the controversy and allegations over gene therapy, clinical research is continuing, and something close to a success story is emerging, particularly in the treatment of hemophilia. Indeed, some of the most encouraging results to date have been reported in this past year. But the field has been irrevocably changed by the 1999 death of Jesse Gelsinger in a gene therapy trial at the University of Pennsylvania in Philadelphia and the stringent regulations that have since emerged. The current environment could deal a hefty blow to a field long plagued by doubt--or simply mark its transition from infancy to maturity.