Studies on hiPSC-derived MNs obtained from ALS patients carrying gain-of-function FUS mutations demonstrated hypoexcitability and axonal transport defects linked to the reduced acetylation of α-tubulin, compared to isogenic controls; the inhibition of HDAC6, the major deacetylating enzyme of α-tubulin, rescued the defects in axonal transport [125]. The gene discussed is FUS; the disease is amyotrophic lateral sclerosis.