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Baby KJ Muldoon becomes first patient treated with personalized CRISPR gene-editing therapy
Children's Hospital of Philadelphia / Penn MedicinePersonalized Base/Prime Editing Platform
At 6 months old, baby KJ received his first infusion of a custom-designed base-editing CRISPR therapy delivered via lipid nanoparticles, the first personalized gene-editing treatment of its kind. The therapy was designed to correct his CPS1 genetic defect, preventing toxic ammonia buildup in his liver.