Nevertheless, ASOs have shown to be efficient and safe in animal models and have led to the initiation of several clinical trials for SOD1-ALS, C9orf72-ALS, and SMN2 for SMA (Miller T. et al., 2013; Meyer et al., 2015; Mendell et al., 2017). Here, C9orf72 is linked to amyotrophic lateral sclerosis.