We assessed the efficacy and selectivity of allele-specific silencing, by injecting lentiviral vectors encoding human mutant ataxin-3(C), together with the corresponding shRNA (shAtaxMUT(C)), into rat striatum. We recently demonstrated that the striatum is affected in MJD, whether in our rat model, transgenic mice or patients [4]. Here, ATXN3 is linked to Machado-Joseph disease.