The trans-activation response DNA-binding protein of 43 kDa (TDP-43) regulates various RNA-processing steps1–3 and is found in the neuropathological lesions of patients with amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD)4,5. The gene discussed is TARDBP; the disease is amyotrophic lateral sclerosis.