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.
journal_name
Neurobiol Disjournal_title
Neurobiology of diseaseauthors
Keiser MS,Geoghegan JC,Boudreau RL,Lennox KA,Davidson BLdoi
10.1016/j.nbd.2013.04.003subject
Has Abstractpub_date
2013-08-01 00:00:00pages
6-13eissn
0969-9961issn
1095-953Xpii
S0969-9961(13)00107-1journal_volume
56pub_type
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