In a landmark achievement for precision medicine, scientists have used a customized gene-editing therapy to save the life of a single infant—ushering in a new era of bespoke genetic treatments that may soon reach more patients.
Late last year, researchers across multiple U.S. institutions raced to design and produce a tailor-made therapy for baby KJ Muldoon, who was born with a fatal mutation preventing his liver from producing a key detoxifying enzyme, carbamoyl phosphate synthetase 1 (CPS1). Without intervention, ammonia would have built up in his blood, causing irreversible brain damage.
