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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 (OTC) deficiency, sponsored by the company Ultragenyx, hasn’t helped everyone with the inherited disease, which causes dangerously high ammonia levels in the blood and affects an estimated one in 50,000 people. Still, there have been no serious safety problems. 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 (ASGCT).
“It’s very heartening that this went well and there were no untoward effects and partial efficacy,”...