Targeting CAG repeat RNAs reduces Huntington’s disease phenotype independently of huntingtin levels

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 stabl...

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發表在:J Clin Invest
Main Authors: Rué, Laura, Bañez-Coronel, Mónica, Creus-Muncunill, Jordi, Giralt, Albert, Alcalá-Vida, Rafael, Mentxaka, Gartze, Kagerbauer, Birgit, Zomeño-Abellán, M. Teresa, Aranda, Zeus, Venturi, Veronica, Pérez-Navarro, Esther, Estivill, Xavier, Martí, Eulàlia
格式: Artigo
語言:Inglês
出版: American Society for Clinical Investigation 2016
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Research Article
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC5096913/
https://ncbi.nlm.nih.gov/pubmed/27721240
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI83185
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