Gene therapy offers hope for children with rare fatal brain disease

This study tested a gene therapy for children with metachromatic leukodystrophy (MLD), a rare and fatal genetic disease that damages the brain and nerves. The treatment involves taking the child's own blood stem cells, adding a working copy of the faulty gene, and giving the cells back after a short course of chemotherapy. The goal was to slow or stop the disease from getting worse. The study included 20 children who were either not yet showing symptoms or had very early signs of the disease.