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Toward RNAi Therapy for the Polyglutamine Disease Machado–Joseph Disease
Machado–Joseph disease (MJD) is a dominantly inherited ataxia caused by a polyglutamine-coding expansion in the ATXN3 gene. Suppressing expression of the toxic gene product represents a promising approach to therapy for MJD and other polyglutamine diseases. We performed an extended therapeutic trial...
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| Asıl Yazarlar: | , , , , , , , , , , , |
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| Materyal Türü: | Artigo |
| Dil: | Inglês |
| Baskı/Yayın Bilgisi: |
Nature Publishing Group
2013
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| Konular: | |
| Online Erişim: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3808129/ https://ncbi.nlm.nih.gov/pubmed/23765441 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.144 |
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