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...

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Veröffentlicht in:	The Journal of clinical investigation 2016
Hauptverfasser:	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, 1955, Martí, Eulàlia
Format:	Artikel
Sprache:	eng
Schlagworte:	Corea de Huntington (Malaltia) Fenotip
Online-Zugang:	Volltext
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