The neuronal ceroid lipofuscinoses, or Batten disease, are a group of fatal inherited neurodegenerative lysosomal storage disorders. Despite having defined genetic causes, the underlying disease mechanisms remain poorly understood and treatments are limited. Cooper and colleagues highlight recent advances in understanding the cell biology and disease pathogenesis, which could inform future therapeutic development.
- Ewa A. Ziółkowska
- Keigo Takahashi
- Jonathan D. Cooper
