TARDBP and amyotrophic lateral sclerosis: Notably, the mouse and human models used in this study do not carry an intermediate-length repeat in their respective ATXN2 genes; thus, we did not determine whether RfxCas13d could suppress TDP-43 aggregation in the context of the polyQ expansion, an important point that will require further study, though we note that the ataxin-2-targeting ASO under evaluation in human trials is being tested in ALS patients both with and without an intermediate-length polyQ expansion (ClinicalTrials.gov Identifier: NCT04494256).