A common pathological feature of these diseases is TAR DNA-binding protein 43 (TDP-43; encoded by TARDBP) mislocalization from the nucleus to the cytoplasm of neurons (Neumann et al., 2006), which occurs in 97% of ALS cases and in 45% of FTLD cases (Ling et al., 2013), and includes both sporadic forms and individuals with mutations in C9orf72 and progranulin genes (DeJesus-Hernandez et al., 2011; Cairns et al., 2007). The gene discussed is TARDBP; the disease is amyotrophic lateral sclerosis.