Metachromatic leukodystrophy (MLD) is a rare, rapidly progressing disorder of the developing nervous system in children. Gene therapy can only slow or halt the progression of the disease if given very early. Dr. Laugwitz’s project aims to use reliable molecular biomarkers to pinpoint the optimal timing of treatment and to assess therapeutic success. The approach analyzes molecular data from children with MLD before and after gene therapy. It also employs patient-derived cell models to study disease mechanisms and potential adjunct therapies.