Personalized Gene Therapy Is Here

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Personalized Gene Therapy Is Here

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In a historic medical breakthrough, Children's Hospital of Philadelphia (CHOP) and Penn Medicine have successfully treated an infant with a custom-designed CRISPR gene-editing therapy. Baby KJ, diagnosed with a rare, fatal metabolic disorder (CPS1 deficiency), received the personalized therapy in early 2025, marking the first time this tailored, in vivo "base editing" technology has been used in a human patient.

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