TDP-43 aggregation is observed in many neurodegenerative disorders,54 including >97% of sporadic and familial ALS patients.55 However, TDP-43 is mutated in 1–4% of the cases.56 This indicates that regulators of TDP-43 are affected in ALS disease, thus allowing TDP-43 to accumulate and aggregate. This evidence concerns the gene TARDBP and amyotrophic lateral sclerosis.