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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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Podrobná bibliografie
Vydáno v:Mol Ther
Hlavní autoři: Ramachandran, Pavitra S, Boudreau, Ryan L, Schaefer, Kellie A, La Spada, Albert R, Davidson, Beverly L
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2014
Témata:
On-line přístup: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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