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Nonallele-specific Silencing of Mutant and Wild-type Huntingtin Demonstrates Therapeutic Efficacy in Huntington's Disease Mice

Huntington's disease (HD) is a fatal neurodegenerative disease caused by mutant huntingtin (htt) protein, and there are currently no effective treatments. Recently, we and others demonstrated that silencing mutant htt via RNA interference (RNAi) provides therapeutic benefit in HD mice. We have...

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Detalhes bibliográficos
Main Authors: Boudreau, Ryan L, McBride, Jodi L, Martins, Inês, Shen, Shihao, Xing, Yi, Carter, Barrie J, Davidson, Beverly L
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2009
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2835182/
https://ncbi.nlm.nih.gov/pubmed/19240687
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.17
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