Doctors successfully treated a baby with the first ever personalized gene-editing therapy

A team of doctors and scientists have successfully treated a rare genetic condition with the first-ever personalized gene-editing therapy. Results of the groundbreaking treatment have been published in The New England Journal of Medicine, with an accompanying editorial by a doctor who had previously overseen the FDA's gene-therapy regulation efforts.

The patient in this historic case was KJ, an infant born with CPS1 deficiency, which has about a 50 percent mortality rate within the first week. Patients that do survive can experience severe brain disease, mental and developmental delays, and potential liver transplants. His care team developed a...