Friedreich’s ataxia (FRDA) is a neurodegenerative disorder driven by frataxin (FXN) deficiency. Here, the authors show that FXN loss in human microglia causes mitochondrial and lysosomal defects that trigger neuroinflammation and neurodegeneration, rescued by gene editing.
- Carla Pernaci
- Avalon Johnson
- Nicole G. Coufal
