Gene therapy provides lasting immune protection for children with ADA-SCID

Gene therapy has shown a 95% success rate in restoring immune function in children with ADA-SCID, offering lasting protection. The experimental treatment involves modifying a child's own stem cells with a healthy ADA gene, which are then reinfused. This approach has maintained immune function long-term without serious complications in the largest study of its kind. This therapy, which has also seen success with cryopreserved cells improving accessibility, is moving towards FDA approval, potentially offering a new standard of care for this rare and fatal disorder.