Therefore, although knock-out mice for ataxin-3 have no major abnormalities [27], [28] and generalized silencing of ataxin-3 in the context of MJD has proved to be safe and effective [29], here we used the more cautious approach of allele-specific silencing for the mutant ataxin-3. This evidence concerns the gene ATXN3 and Spinocerebellar ataxia type 3.