Health

Pioneering Gene Therapy Trial for Genetic Disease

Updated 2026-05-20 19:05 UTC 1 source Positive

A team from UCSF has submitted an application to the FDA seeking approval for a small trial of in utero gene therapy for treating a rare lysosomal storage disorder. This pioneering approach aims to treat genetic diseases before birth.

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STAT News
A UCSF team has submitted an application to the FDA seeking approval for a small trial of in utero gene therapy for a rare lysosomal storage disorder.
2026-05-20 18:48 UTC
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