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 … ⌘ Read more