Genetic alterations in RNA-binding proteins can lead to genetic diseases, including amyotrophic lateral sclerosis caused by fused-in-sarcoma (FUS)/TAR DNA binding protein-43 (TDP-43) mutations, and myelodysplastic syndromes caused by U2AF35/ splicing factor 3b subunit 1 (SF3B1) mutations [192]. The gene discussed is TARDBP; the disease is myelodysplastic syndrome.