One-Time gene therapy tested to halt devastating childhood brain disease

This study tested a one-time gene therapy called OTL-200 in young children with a rare, severe genetic brain disease called early-onset metachromatic leukodystrophy (MLD). The therapy involved collecting a child's own blood stem cells, correcting their genetic defect in a lab, and then returning the cells to try to stop or slow the disease. The main goals were to see if the treatment was safe and if it could help preserve the children's ability to move and function.