Own stem cells Gene-Hacked to fight rare hurler syndrome

This study tests a one-time gene therapy for children with Hurler syndrome, a severe genetic disorder. Doctors take the child's own blood stem cells, add a working copy of the missing gene, and put them back. The goal is to help the body produce the enzyme it lacks, potentially stopping disease damage. The trial checks safety and whether the treatment works in up to 8 children aged 28 days to 11 years.