Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients.

Huntington disease (HD) is an inherited, fatal neurodegenerative disorder caused by a CAG repeat expansion in the huntingtin gene. The mutant protein causes neuronal dysfunction and degeneration resulting in motor dysfunction, cognitive decline, and psychiatric disturbances. Currently, there is no d...

Ausführliche Beschreibung

Bibliografische Detailangaben

Hauptverfasser:	Niels H Skotte, Amber L Southwell, Michael E Østergaard, Jeffrey B Carroll, Simon C Warby, Crystal N Doty, Eugenia Petoukhov, Kuljeet Vaid, Holly Kordasiewicz, Andrew T Watt, Susan M Freier, Gene Hung, Punit P Seth, C Frank Bennett, Eric E Swayze, Michael R Hayden
Format:	Artigo
Sprache:	Inglês
Veröffentlicht:	Public Library of Science (PLoS) 2014-01-01
Schriftenreihe:	PLoS ONE
Online-Zugang:	http://europepmc.org/articles/PMC4160241?pdf=render
Tags:	Tag hinzufügen Keine Tags, Fügen Sie das erste Tag hinzu!