Summary
Twenty-two years ago, one of scientists' first attempts at gene therapy ended in tragedy when a young man died. The story of Jesse Gelsinger, who had a rare liver disorder, became a textbook example of irresponsible medical research. For years, the case hobbled efforts to treat diseases by adding new DNA to a sick person's cells. Now, a fresh effort to cure Gelsinger's disease is bearing fruit, in the latest sign of the field's resurgence. The small, ongoing trial for ornithine transcarbamylase deficiency hasn't helped everyone with the inherited disease, which causes dangerously high ammonia levels in the blood. Still, there have been no serious safety problems. And many of the 11 participants have been able to relax dietary restrictions and drop medications, including three who no longer need those measures at all, researchers reported last week at the virtual meeting of the American Society of Gene & Cell Therapy.
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May 21, 2021 at 12:54AM
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Gene therapy that once led to tragedy scores success - Science
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