LOS ANGELES, May 15 (Xinhua) -- In a groundbreaking medical milestone, U.S. researchers have successfully developed and delivered a personalized gene-editing therapy to treat an infant suffering from a rare and life-threatening genetic disorder.
The child, diagnosed shortly after birth with carbamoyl phosphate synthetase 1 (CPS1) deficiency, became the first human to receive a customized CRISPR-based treatment targeting the root cause of the condition, according to a release of the U.S. National Institutes of Health (NIH) on Thursday.
