Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic de...
Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi
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United States: National Academy of Sciences
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English
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United States: National Academy of Sciences
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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 mutant human HD transgenes improved neuropathology and be...
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Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi
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TN_cdi_proquest_miscellaneous_20668365
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_20668365
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ISSN
0027-8424,1091-6490
E-ISSN
1091-6490
DOI
10.1073/pnas.0801775105