TARDBP and amyotrophic lateral sclerosis: TDP-43 has been identified as the major disease protein aggregating in cytoplasmic inclusions that characterize both ALS and FTLD, and mutations in the encoding TARDBP gene have been found in familial cases (7,8), strongly suggesting that both cytoplasmic inclusions and loss of function of TDP-43 are causally related to disease formation (9–11).