World's First Personalized Gene Editing Heals Baby with Rare Genetic Disorder

A Philadelphia-based pediatrician, Dr. Rubin, recently shared a video detailing the successful treatment of a nine-and-a-half-month-old baby boy, KJ, using the world's first personalized gene editing therapy. KJ suffered from CPS1 deficiency, a rare genetic disorder that disrupts the urea cycle, leading to a toxic buildup of ammonia. This condition can cause severe neurological damage and even death. Dr. Rubin explains in his video, "What is so exciting is that there are so many prospects for this type of technology to help all of these different rare genetic disorders." The successful treatment represents a significant advancement in gene editing technology and offers hope for families affected by similar rare genetic disorders. The research leading to this breakthrough was funded by the NIH (National Institutes of Health), highlighting the importance of public investment in medical research.