ATXN3 and nervous system disorder: In particular, the positive effects observed for antisense oligonucleotide therapies targeting ATXN2 expression, together with the efficacy demonstrated by AAV5-miATXN3 vectors in SCA3 cellular models, could provide a foundation for the clinical translation of RNAi-based therapies, potentially extendable to polyglutamine disorders and other neurological diseases due to protein aggregation [88,120].