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NIH/Office of the Director


Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment

A team of researchers has successfully treated an infant with a life-threatening, incurable genetic disease using personalized gene editing therapy. The infant, who was diagnosed shortly after birth, showed positive responses to the treatment and improved symptoms over time.

SourceNIH/Office of the Director·JournalNew England Journal of Medicine·TypeRandomized controlled/clinical trial·DateMay 15, 2025