Therefore, in the present study we used lentiviral vectors (LV) encoding short-hairpin RNAs (shRNAs) targeting this SNP, to downregulate mutant ataxin-3 in the cerebellum of a transgenic MJD mouse model that exhibits an early and very severe motor and neuropathological phenotype [15]. The gene discussed is ATXN3; the disease is Spinocerebellar ataxia type 3.