This model was generated in a C57/BL6 background, by the introduction of a truncated form of human ataxin-3 with 69 repeats [15], [27] and encoding the C variant of the human ataxin-3 transgene sequence that is present in 70% of the MJD patients [15], [25], [28], making this model particularly adequate for the allele-specific silencing here performed. This evidence concerns the gene ATXN3 and Machado-Joseph disease.