ASOs targeting disease-causative genes associated with ALS/FTD, including SOD1, C9orf72, ataxin-2, and FUS, have already been demonstrated to show therapeutic effects in preclinical evaluations and are now being clinically tested in patients, holding great promise for the development of ASO-based therapies for ALS/FTD in the near future. The gene discussed is ATXN2; the disease is amyotrophic lateral sclerosis.