In order to identify new therapeutic strategies for FTD and ALS, recent studies have explored the use of antisense oligonucleotides (ASOs) or pharmacologic inhibitors of histone deacetylase 6 (HDAC6) to rescue axonal transport defects in induced pluripotent stem cell (iPSC)-derived motor neurons from familial ALS patients (Guo et al., 2017; Fazal et al., 2021). The gene discussed is HDAC6; the disease is amyotrophic lateral sclerosis.