TDP-43 that forms cytoplasmic aggregates in neurons of amyotrophic lateral sclerosis (ALS), AD, and frontotemporal lobar degeneration (FTLD) patients [20] also shows altered expression and mislocalization in the Niemann-Pick type C mouse and in a human neuronal model of the disease [21]. Here, TARDBP is linked to amyotrophic lateral sclerosis.