A decade after gene therapy, children born with deadly immune disorder remain healthy

Photo: Evangelina Vaccaro, above, who received the gene therapy for ADA-SCID in a clinical trial in 2012. Photo courtesy of Alysia Padilla-Vaccaro. Article by Sarah C.P. Williams. UCLA Newsroom – October 15, 2021.

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. […]

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Curated by John Wysocki

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