Amyotrophic lateral sclerosis (ALS) is a devastating and rapid fatal adult-onset neurodegenerative disease with a defining neuropathological feature of altered expression and function of transactive response DNA-binding protein 43 (TDP-43) in motor neurons of the brain and spinal cord [7, 31, 43, 48]. Here, TARDBP is linked to amyotrophic lateral sclerosis.