16
views
0
recommends
+1 Recommend
0 collections
    0
    shares
      • Record: found
      • Abstract: found
      • Article: not found

      RNAi or overexpression: alternative therapies for Spinocerebellar Ataxia Type 1.

      Neurobiology of Disease
      Animals, Behavior, Animal, physiology, Blotting, Western, Brain, pathology, Dependovirus, genetics, Gait, Genetic Vectors, HEK293 Cells, Humans, Immunohistochemistry, Immunoprecipitation, In Situ Hybridization, Locomotion, Mice, Mice, Transgenic, MicroRNAs, biosynthesis, Nerve Tissue Proteins, Nuclear Proteins, Plasmids, Postural Balance, RNA Interference, RNA, Small Interfering, therapeutic use, Real-Time Polymerase Chain Reaction, Spinocerebellar Ataxias, psychology, therapy

      Read this article at

      ScienceOpenPublisherPMC
      Bookmark
          There is no author summary for this article yet. Authors can add summaries to their articles on ScienceOpen to make them more accessible to a non-specialist audience.

          Abstract

          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 ataxin-1 expression (miS1). Delivery of either ataxin-1-like or miS1 viral vectors to SCA1 mice cerebella resulted in widespread cerebellar Purkinje cell transduction and improved behavioral and histological phenotypes. Our data indicate the utility of either approach as a possible therapy for SCA1 patients. Published by Elsevier Inc.

          Related collections

          Author and article information

          Comments

          Comment on this article