Disease-modifying therapies designed or formulated to specifically target the ATXN2 gene, including the use of antisense oligonucleotides, are currently being studied as a promising therapeutic approach for ALS (Van Den Heuvel et al., 2014; Scoles and Pulst, 2018; Hergesheimer et al., 2020). The gene discussed is ATXN2; the disease is amyotrophic lateral sclerosis.