Silencing Mutant Ataxin-3 Rescues Motor Deficits and Neuropathology in Machado-Joseph Disease Transgenic Mice

Machado-Joseph disease (MJD) or spinocerebellar ataxia type 3 (SCA3) is an autosomal dominantly-inherited neurodegenerative disorder caused by the over-repetition of a CAG codon in the MJD1 gene. This expansion translates into a polyglutamine tract that confers a toxic gain-of-function to the mutant...

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Main Authors: Nóbrega, Clévio, Nascimento-Ferreira, Isabel, Onofre, Isabel, Albuquerque, David, Hirai, Hirokazu, Déglon, Nicole, de Almeida, Luís Pereira
Format: Artigo
Sprog:Inglês
Udgivet: Public Library of Science 2013
Fag:
Research Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3551966/
https://ncbi.nlm.nih.gov/pubmed/23349684
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0052396
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