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Donald J. Musk@lemmy.todayM to Science News@lemmy.todayEnglish · 2 months ago

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

www.sciencedaily.com

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

www.sciencedaily.com

Donald J. Musk@lemmy.todayM to Science News@lemmy.todayEnglish · 2 months ago
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In a stunning medical breakthrough, scientists used customized CRISPR gene editing to save a baby’s life from a rare, previously incurable liver disorder—CPS1 deficiency—just six months after birth. This marks the first time a tailored gene therapy has been safely delivered to a human patient, rewriting the infant's DNA directly inside liver cells.
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