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Broad Therapeutic Benefit After RNAi Expression Vector Delivery to Deep Cerebellar Nuclei: Implications for Spinocerebellar Ataxia Type 1 Therapy
Spinocerebellar ataxia type 1 (SCA1) is an autosomal dominant, late-onset neurodegenerative disease caused by a polyglutamine (polyQ) expansion in the ataxin-1 protein, which causes progressive neurodegeneration in cerebellar Purkinje cells and brainstem nuclei. Here, we tested if reducing mutant at...
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| Main Authors: | , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Nature Publishing Group
2014
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| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3944323/ https://ncbi.nlm.nih.gov/pubmed/24419082 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.279 |
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