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Unexpected off-targeting effects of anti-huntingtin ribozymes and siRNA in vivo
Gene transfer strategies to reduce levels of mutant huntingtin (mHtt) mRNA and protein by targeting human Htt have shown therapeutic promise in vivo. Previously, we have reported that a specific, adeno-associated viral vector (rAAV)-delivered short-hairpin RNA (siHUNT-2) targeting human Htt mRNA une...
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| Главные авторы: | , , , |
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| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
2007
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| Предметы: | |
| Online-ссылка: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2695881/ https://ncbi.nlm.nih.gov/pubmed/18166484 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.nbd.2007.11.003 |
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