News for Healthier Living

Improving Access to Cell and Gene Therapy in Pediatrics

Nearly 30 years ago, Children's Hospital Los Angeles supported one of the first clinical trials for a pioneering stem cell gene therapy for children with a life-threatening form of severe combined immunodeficiency (SCID). Called ADA-SCID--a type of "bubble boy disease" caused by mutations in the adenosine deaminase (ADA) gene--this rare disorder leaves babies with virtually no immune system.

October 9, 2024


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