In recent years, ASOs targeting sense RNAs have successfully been tested on mouse and human cellular models, showing a decrease of toxic transcripts and a partial rescue of the disease phenotype, as in SCA2 [217,218], SCA7 [219], HD [220], DM1 [221] and C9ORF72 FTD/ALS [222,223,224,225]. The gene discussed is ATXN2; the disease is amyotrophic lateral sclerosis.