Preclinical data obtained in murine or iPSC models of the major familial ALS subtypes revealed axonal transport defects due to pathogenic variants in the genes encoding for FUS [431-433], SOD1 [434-436], TARDBP [437-439] and C9orf72 [440-443]. The gene discussed is C9orf72; the disease is amyotrophic lateral sclerosis.