The trans-activating response region DNA-binding protein 43 (TARDBP, TDP-43) is a product of a causative gene for amyotrophic lateral sclerosis (ALS), and its disease-associated mutations cause its accumulation in the cytoplasm of cells in ALS-related lesions in the brain (1,2). The gene discussed is TARDBP; the disease is amyotrophic lateral sclerosis.