An exciting step forward in ALS genetics is represented by the recent discovery of mutations in TDP-43 (encoded by TARDBP) [16] and the related RNA-binding protein fused in sarcoma/translocated in liposarcoma (FUS) [17] in familial and sporadic cases that has shifted the focus of much research on RNA metabolism, and implicated abnormal RNA processing in ALS pathogenesis. This evidence concerns the gene TARDBP and amyotrophic lateral sclerosis.