Gene therapy that once led to tragedy scores success.
Twenty-two years ago, one of scientists9 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 person9s cells. Now, a fresh effort to cure Gelsinger9s disease is bearing fruit, in the latest sign of the field9s resurgence. The small, ongoing trial for ornithine transcarbamylase deficiency, hasn9t 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.