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RNAi prevents and reverses phenotypes induced by mutant human ataxin‐1
OBJECTIVE: Spinocerebellar ataxia type 1 is an autosomal dominant fatal neurodegenerative disease caused by a polyglutamine expansion in the coding region of ATXN1. We showed previously that partial suppression of mutant ataxin‐1 (ATXN1) expression, using virally expressed RNAi triggers, could preve...
Autores principales: | , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2016
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5115960/ https://www.ncbi.nlm.nih.gov/pubmed/27686464 http://dx.doi.org/10.1002/ana.24789 |