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A few weeks ago we introduced you to KJ, who made medical history as the first patient to receive a personalized gene editing therapy. After almost a year at CHOP, KJ is now at home with his family. 🎓 Tune in to NBC's Today Show tomorrow morning to hear from his family! | Children's Hospital of Philadelphia
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FacebookChildren's Hospital of Philadelphia
KJ Muldoon, the infant with a life-threatening metabolic disorder who received a pioneering gene-editing therapy at Children’s Hospital of Philadelphia, went home Tuesday after 307 days at the hospital, CHOP announced. KJ risked irreparable brain damage from his condition, called severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, in which his liver could not process protein. CPS1 deficiency is deadly in more than half of cases. Doctors at CHOP rapidly developed within six months an exper
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