While mutations observed in the TARDBP gene are rare (<1%), the protein encoded by the TARDBP gene—a TDP43 protein—forms cytoplasmic aggregates in approximately 97% of ALS cases including the genetic forms of ALS (exceptions are SOD1-ALS and FUS-ALS), making it hugely relevant for understanding the pathophysiology of ALS [18]. The gene discussed is FUS; the disease is amyotrophic lateral sclerosis.