BIN1 reduction ameliorates DNM2-related Charcot–Marie–Tooth neuropathy

Proceedings of the National Academy of Sciences, Volume 122, Issue 10, March 2025.
SignificanceHeterozygous mutations in theDNM2gene can cause Charcot–Marie–Tooth (CMT) neuropathy, for which no therapies currently exist. Previous studies have shown that increasing BIN1 (amphiphysin 2) can rescue centronuclear myopathy, another disease …