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Universal RNAi Triggers for the Specific Inhibition of Mutant Huntingtin, Atrophin-1, Ataxin-3, and Ataxin-7 Expression
The expansion of CAG repeats within the coding region of associated genes is responsible for nine inherited neurodegenerative disorders including Huntington’s disease (HD), spinocerebellar ataxias (SCAs), and dentatorubral-pallidoluysian atrophy (DRPLA). Despite years of research aimed at developing...
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| Veröffentlicht in: | Mol Ther Nucleic Acids |
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| Hauptverfasser: | , , |
| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
American Society of Gene & Cell Therapy
2019
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| Schlagworte: | |
| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6957814/ https://ncbi.nlm.nih.gov/pubmed/31927329 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2019.12.012 |
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