ASOs have also been used to successfully alleviate symptoms in various model systems of ALS, including C9orf72 patient-induced pluripotent stem cell (iPSC) neurons, and TDP-43 and C9orf72 mouse models (Donnelly et al., 2013; Sareen et al., 2013; Jiang et al., 2016; Becker et al., 2017; Scoles et al., 2017), indicating their therapeutic potential. This evidence concerns the gene C9orf72 and amyotrophic lateral sclerosis.