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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...

詳細記述

保存先:
書誌詳細
出版年:Mol Ther
主要な著者: Ramachandran, Pavitra S, Boudreau, Ryan L, Schaefer, Kellie A, La Spada, Albert R, Davidson, Beverly L
フォーマット: Artigo
言語:Inglês
出版事項: Nature Publishing Group 2014
主題:
オンライン・アクセス: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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