World’s First Custom Gene-Editing Treatment Saves Baby With Rare Disease

A baby named KJ has become the first person to receive a personalized gene-editing treatment, marking a historic moment in medicine. Born with CPS1 deficiency, a rare condition that can be fatal within days, KJ was treated at Children’s Hospital of Philadelphia using CRISPR-based base editing tailored to his unique mutation. After three doses, KJ is thriving, meeting milestones and preparing to go home. The success highlights the potential for individualized gene therapies to treat thousands of rare genetic diseases, supported by decades of federally funded research.

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Source: NYTimes