The concomitant occurrence of reduced ADAR2 levels and TDP-43 pathology in the same motor neurons (Aizawa et al, 2010) and the presence of calpain-dependent abnormal TDP-43 fragments in the brains and spinal cords of sporadic ALS patients (Yamashita et al, 2012a) suggest that the molecular cascade observed in AR2 mice is similar to what occurs in the motor neurons of ALS patients and that normalization of ADAR2 activity represents a potential therapeutic strategy for ALS patients (Aizawa et al, 2010; Yamashita et al, 2012a, 2012b). The gene discussed is TARDBP; the disease is amyotrophic lateral sclerosis.