Targeting CAG repeat RNAs reduces Huntington's disease phenotype independently of huntingtin levels
Targeting CAG repeat RNAs reduces Huntington's disease phenotype independently of huntingtin levels
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United States: American Society for Clinical Investigation
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Language
English
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Publisher
United States: American Society for Clinical Investigation
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Huntington's disease (HD) is a polyglutamine disorder caused by a CAG expansion in the Huntingtin (HTT) gene exon 1. This expansion encodes a mutant protein whose abnormal function is traditionally associated with HD pathogenesis; however, recent evidence has also linked HD pathogenesis to RNA stable hairpins formed by the mutant HTT expansion. Her...
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Full title
Targeting CAG repeat RNAs reduces Huntington's disease phenotype independently of huntingtin levels
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TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_5096913
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_5096913
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ISSN
0021-9738
E-ISSN
1558-8238
DOI
10.1172/JCI83185