Nonallele Specific Silencing of Ataxin-7 Improves Disease Phenotypes in a Mouse Model of SCA7

Spinocerebellar ataxia type 7 (SCA7) is a late-onset neurodegenerative disease characterized by ataxia and vision loss with no effective treatments in the clinic. The most striking feature is the degeneration of Purkinje neurons of the cerebellum caused by the presence of polyglutamine-expanded atax...

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Detalles Bibliográficos
Publicado en:Mol Ther
Main Authors: Ramachandran, Pavitra S, Boudreau, Ryan L, Schaefer, Kellie A, La Spada, Albert R, Davidson, Beverly L
Formato: Artigo
Idioma:Inglês
Publicado: Nature Publishing Group 2014
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Original Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC4435484/
https://ncbi.nlm.nih.gov/pubmed/24930601
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2014.108
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