Baby's Gene Editing Shows Transformative Results | JQJO

Six-month-old KJ, suffering from a genetic liver disease, received a groundbreaking gene-editing therapy. Researchers rapidly secured FDA approval for a customized treatment, successfully tested in mice and monkeys. The therapy corrected his genetic mutation, allowing him to tolerate more protein and significantly improve his weight. While still requiring monitoring, KJ's progress is deemed transformative, and he is preparing for his first discharge from the hospital.