A decade after gene therapy, children born with deadly immune disorder remain healthy
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15 October 2021A decade after gene therapy, children born with deadly immune disorder remain healthy
Over a decade ago, UCLA physician-scientists began using a pioneering gene therapy they developed to treat children born with a rare and deadly immune system disorder. They now report that the effects of the therapy appear to be long-lasting, with 90% of patients who received the treatment eight to 11 years ago still disease-free.
