Baby gets world's first personalised gene therapy treatment
undefined 21, 2025
A 9-month-old baby with a rare genetic disease, CPS1 deficiency, is “growing and thriving” after receiving a bespoke gene-editing therapy. Developed in just seven months using CRISPR, the treatment fixes the exact DNA mutation causing his condition. Since treatment, KJ Muldoon has improved eating, recovered better from illnesses, and needs fewer meds. This first-of-its-kind therapy marks a major step toward curing rare genetic disorders and could transform personalized medicine soon.
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