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

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...

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Bibliographic Details
Main Authors: 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: Article
Language:English
Published: Public Library of Science (PLoS) 2014-01-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC4160241?pdf=render

Internet

http://europepmc.org/articles/PMC4160241?pdf=render

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