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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...

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Hlavní autoři: Nóbrega, Clévio, Nascimento-Ferreira, Isabel, Onofre, Isabel, Albuquerque, David, Déglon, Nicole, Pereira de Almeida, Luís
Médium: Artigo
Jazyk:Inglês
Vydáno: Public Library of Science 2014
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4140724/
https://ncbi.nlm.nih.gov/pubmed/25144231
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0100086
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