RNAi or overexpression: Alternative therapies for Spinocerebellar Ataxia Type 1

  title={RNAi or overexpression: Alternative therapies for Spinocerebellar Ataxia Type 1},
  author={Megan S. Keiser and James C. Geoghegan and Ryan L. Boudreau and Kim A. Lennox and Beverly L. Davidson},
  journal={Neurobiology of Disease},
Spinocerebellar Ataxia Type 1 (SCA1) is an autosomal dominant late onset neurodegenerative disease caused by an expanded polyglutamine tract in ataxin-1. Here, we compared the protective effects of overexpressing ataxin-1-like using recombinant AAVs, or reducing expression of mutant ataxin-1 using virally delivered RNA interference (RNAi), in a transgenic mouse model of SCA1. For the latter, we used an artificial microRNA (miR) design that optimizes potency, efficacy and safety to suppress… CONTINUE READING


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