Abnormal aggregation of 43-kDa trans-activation response (TAR) DNA-binding protein (TDP-43) is a hallmark pathological feature in nearly all ALS patients, except for patients carrying mutations in the superoxide dismutase 1 (SOD1) and fused in sarcoma (FUS) genes [1]. The gene discussed is FUS; the disease is amyotrophic lateral sclerosis.