Allele-specific silencing of mutant huntingtin in rodent brain and human stem cells.

Allele-specific silencing of mutant huntingtin in rodent brain and human stem cells.

Huntington's disease (HD) is an autosomal dominant neurodegenerative disorder resulting from polyglutamine expansion in the huntingtin (HTT) protein and for which there is no cure. Although suppression of both wild type and mutant HTT expression by RNA interference is a promising therapeutic st...

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Bibliographic Details
Main Authors: Valérie Drouet, Marta Ruiz, Diana Zala, Maxime Feyeux, Gwennaëlle Auregan, Karine Cambon, Laetitia Troquier, Johann Carpentier, Sophie Aubert, Nicolas Merienne, Fany Bourgois-Rocha, Raymonde Hassig, Maria Rey, Noëlle Dufour, Frédéric Saudou, Anselme L Perrier, Philippe Hantraye, Nicole Déglon
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2014-01-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC4057216?pdf=render

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http://europepmc.org/articles/PMC4057216?pdf=render

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