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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...
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| Publicado en: | Ann Neurol |
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| Main Authors: | , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado: |
John Wiley and Sons Inc.
2016
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| Assuntos: | |
| Acceso en liña: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5115960/ https://ncbi.nlm.nih.gov/pubmed/27686464 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ana.24789 |
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