In the transgenic TDP43 ALS murine model, anti-ATXN2 ASOs rescued the phenotype and prolonged survival, particularly when given early.23A phase 1 clinical trial is ongoing to assess the safety and efficacy of IT anti-ATXN2 ASOs in patients with sporadic and ATXN2-related ALS.21 This evidence concerns the gene TARDBP and amyotrophic lateral sclerosis.