Cytoplasmic aggregated deposits of the nuclear TAR DNA-binding protein 43 (TDP-43) characterize neurons in most amyotrophic lateral sclerosis (ALS) and approximately half of frontotemporal lobar degeneration (FTLD) cases, now collectively called TDP-43 proteinopathies [1,2,3]. The gene discussed is TARDBP; the disease is amyotrophic lateral sclerosis.