Open access
Jul 2026
HDAC6 inhibition alleviates mitochondrial trafficking in models of Charcot-Marie-Tooth disease type 2A
A variant-specific insight is revealed into CMT2A disease mechanisms and HDAC6 is confirmed as a promising target for further therapeutic development by showing that mitochondrial trafficking defects could be alleviated by treatment with an HDAC6 inhibitor.
Lydia H. Jestice, Larissa Butler, Rebecca A. Lea et al.
· JCI Insight · 0 citations