One-off gene therapy for rare immune disease still effective 10 years later

Evangelina Vaccaro, above, received the gene therapy for a rare immune disease in 2012, and is still feeling the positive effects a decade later

A long-term follow-up study tracking a group of children who received experimental gene therapy as part of an early-stage clinical trial is reporting the treatment is safe and effective up to 10 years later. The research offers rare insight into the long-lasting effects of a yet-to-be-approved gene therapy.

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Category: Science

Tags: gene therapy, The Immune System, Genetics, UCLA

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Anthony Harris