Scientists test 'One-Time' gene fix for devastating childhood disease

This is a first-in-human safety study testing a new gene therapy for young boys with a severe form of Hunter syndrome, a rare genetic disorder. Researchers will collect a child's own stem cells, add a working copy of a missing gene in the lab, and return the cells to the child's body. The goal is to see if the body can then produce the missing enzyme itself, which may help treat symptoms, including those affecting the brain. Five young patients will be closely followed for at least two years to check safety.