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
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