NIH News Releases

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

NIH-supported gene-editing platform lays groundwork to rapidly develop treatments for other rare genetic diseases.

May 15, 2025 - 21:22
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Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment
NIH-supported gene-editing platform lays groundwork to rapidly develop treatments for other rare genetic diseases.
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