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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Bibliographic Details
Main Authors: Clévio Nóbrega, Isabel Nascimento-Ferreira, Isabel Onofre, David Albuquerque, Nicole Déglon, Luís Pereira de Almeida
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2014-01-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC4140724?pdf=render