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Therapeutic impact of systemic AAV-mediated RNA interference in a mouse model of myotonic dystrophy
RNA interference (RNAi) offers a promising therapeutic approach for dominant genetic disorders that involve gain-of-function mechanisms. One candidate disease for RNAi therapy application is myotonic dystrophy type 1 (DM1), which results from toxicity of a mutant mRNA. DM1 is caused by expansion of...
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| Publicado no: | Hum Mol Genet |
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| Main Authors: | , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Oxford University Press
2015
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4527493/ https://ncbi.nlm.nih.gov/pubmed/26082468 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddv219 |
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