SOD1 and amyotrophic lateral sclerosis: Although most ALS cases are sporadic, approximately 10% of cases are familial (FALS) and predominantly associated with Mendelian-inherited mutations in genes including Cu/Zn superoxide dismutase (SOD1) and C9ORF72. Notably, the iPSC-derived motor neurons from patients with FALS carrying C9ORF72 hexanucleotide repeat expansion or G93A SOD1 mutation exhibit retromer deficiency, and retromer stabilization by chemical chaperone attenuated the locomotive activity and motor neuron loss in G93A SOD1 Tg mice (Aoki et al., 2017; Muzio et al., 2020).