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Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi

Huntington's disease (HD) is a fatal, dominant neurodegenerative disease caused by a polyglutamine repeat expansion in exon 1 of the HD gene, which encodes the huntingtin protein. We and others have shown that RNAi is a candidate therapy for HD because expression of inhibitory RNAs targeting mu...

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Bibliografiske detaljer
Main Authors: McBride, Jodi L., Boudreau, Ryan L., Harper, Scott Q., Staber, Patrick D., Monteys, Alex Mas, Martins, Inâs, Gilmore, Brian L., Burstein, Haim, Peluso, Richard W., Polisky, Barry, Carter, Barrie J., Davidson, Beverly L.
Format: Artigo
Sprog:Inglês
Udgivet: National Academy of Sciences 2008
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Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2311380/
https://ncbi.nlm.nih.gov/pubmed/18398004
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0801775105
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