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Allele-specific silencing of mutant Ataxin-7 in SCA7 patient-derived fibroblasts
Polyglutamine (polyQ) disorders are inherited neurodegenerative conditions defined by a common pathogenic CAG repeat expansion leading to a toxic gain-of-function of the mutant protein. Consequences of this toxicity include activation of heat-shock proteins (HSPs), impairment of the ubiquitin-protea...
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| Publicado no: | Eur J Hum Genet |
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| Main Authors: | , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Nature Publishing Group
2014
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4231408/ https://ncbi.nlm.nih.gov/pubmed/24667781 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/ejhg.2014.39 |
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