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RNA Interference Mitigates Motor and Neuropathological Deficits in a Cerebellar Mouse Model of Machado-Joseph Disease
Machado-Joseph disease or Spinocerebellar ataxia type 3 is a progressive fatal neurodegenerative disorder caused by the polyglutamine-expanded protein ataxin-3. Recent studies demonstrate that RNA interference is a promising approach for the treatment of Machado-Joseph disease. However, whether gene...
Autores principales: | Nóbrega, Clévio, Nascimento-Ferreira, Isabel, Onofre, Isabel, Albuquerque, David, Déglon, Nicole, Pereira de Almeida, Luís |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Public Library of Science
2014
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4140724/ https://www.ncbi.nlm.nih.gov/pubmed/25144231 http://dx.doi.org/10.1371/journal.pone.0100086 |
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