Silencing Mutant ATXN3 Expression Resolves Molecular Phenotypes in SCA3 Transgenic Mice

Spinocerebellar ataxia type 3 (SCA3) is a neurodegenerative disease caused by a polyglutamine expansion in the deubiquitinating enzyme, Ataxin-3. Currently, there are no effective treatments for this fatal disorder but studies support the hypothesis that reducing mutant Ataxin-3 protein levels might...

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Autores principales: Rodríguez-Lebrón, Edgardo, Costa, Maria doCarmo, Luna-Cancalon, Katiuska, Peron, Therese M, Fischer, Svetlana, Boudreau, Ryan L, Davidson, Beverly L, Paulson, Henry L
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group 2013
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Original Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3808130/
https://ncbi.nlm.nih.gov/pubmed/23820820
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.152
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