Pelizaeus-Merzbacher disease is a pediatric leukodystrophy causing oligodendrocyte cell death. Nobuta et al. show that mutations in human PLP1 gene cause iron-induced cell death through lipid peroxidation, abnormal iron metabolism, and hypersensitivity to free iron. Iron chelation rescues cell death, offering a therapeutic direction for a disease without current treatments.
All Science Journal Classification (ASJC) codes
- Molecular Medicine
- Cell Biology
- gene correction
- induced pluripotent stem cells
- iron chelation
- patient models