Baby Is Healed With World's First Personalized Gene-Editing Treatment

Scientists have successfully treated a 9.5-month-old boy with an ultra-rare genetic disorder using the world's first personalized gene-editing therapy. The patient, identified as KJ, has CPS1 deficiency -- a condition affecting just one in 1.3 million babies that prevents proper ammonia processing and is often fatal.
The breakthrough treatment, detailed in the New England Journal of Medicine, uses base editing technology to correct KJ's specific DNA mutation. The therapy delivers CRISPR componen