In this study, to explore the outcome of mutant ataxin-3 gene silencing in MJD motor phenotype and neuropathology, lentiviral vectors encoding RNA interference transcripts were co-injected with the human full-length mutant ataxin-3 in the cerebellum of wild-type mice (Figure S1 in File S1). The gene discussed is ATXN3; the disease is Machado-Joseph disease.