Artificial miRNAs Targeting Mutant Huntingtin Show Preferential Silencing In Vitro and In Vivo
Artificial miRNAs Targeting Mutant Huntingtin Show Preferential Silencing In Vitro and In Vivo
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United States: Elsevier Inc
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English
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United States: Elsevier Inc
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Huntington's disease (HD) is a dominantly inherited neurodegenerative disease caused by CAG repeat expansion in exon 1 of huntingtin (HTT). Studies in mouse models of HD with a regulated mutant transgene show that continuous mutant allele expression is required for behavioral and pathological signs; when mutant HTT expression declined, neuronal deg...
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Artificial miRNAs Targeting Mutant Huntingtin Show Preferential Silencing In Vitro and In Vivo
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TN_cdi_doaj_primary_oai_doaj_org_article_6cfa4af9168a4d46b7bb2e1c9dab5b25
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_6cfa4af9168a4d46b7bb2e1c9dab5b25
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ISSN
2162-2531
E-ISSN
2162-2531
DOI
10.1038/mtna.2015.7
