ATXN2 and amyotrophic lateral sclerosis: Gene therapy involves the use of ASOs to silence the repeat expansion of GGGGCC of C9ORF72 and target ATXN2; however, in addition to the direct targeting of ALS-associated genetic mutations, it is possible to intervene at the post-transcriptional or post-translational level modulating the expression or function of specific RNAs (sense or antisense) or proteins implicated in ALS.