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Optimization of trans-Splicing for Huntington's Disease RNA Therapy
Huntington's disease (HD) is a devastating neurodegenerative disorder caused by a polyglutamine (polyQ) expansion in exon 1 of the Huntingtin (HTT) gene. We have previously demonstrated that spliceosome-mediated trans-splicing is a viable molecular strategy to specifically reduce and repair mut...
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| 出版年: | Front Neurosci |
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| 主要な著者: | , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
Frontiers Media S.A.
2017
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5641306/ https://ncbi.nlm.nih.gov/pubmed/29066943 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3389/fnins.2017.00544 |
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