About 10% of ALS patients have a genetically inherited form associated with mutations in Cu/Zn superoxide dismutase (SOD1)1–3, TAR DNA binding protein 43 (TDP-43)4,5, and a hexanucleotide repeat expansion of the C9ORF72 gene6,7. The gene discussed is TARDBP; the disease is amyotrophic lateral sclerosis.