Owing to the central role of mutant ataxin-7 in the pathophysiology of SCA7, the current therapeutic approaches are mainly aimed to suppress the mutant protein expression at transcriptional level using RNA interference and antisense oligonucleotides [25], or to proteolytically eliminate mutant ataxin-7 aggregates using drugs with the ability to activate the ubiquitin–proteasome and/or autophagy systems [26]. This evidence concerns the gene ATXN7 and spinocerebellar ataxia 7.