Article
Allele-specific silencing of mutant huntingtin in rodent brain and human stem cells.
PloS one - 1 Jan 2014
Drouet Valérie, Ruiz Marta, Zala Diana, Feyeux Maxime, Auregan Gwennaëlle, Cambon Karine, Troquier Laetitia, Carpentier Johann, Aubert Sophie, Merienne Nicolas, Bourgois-Rocha Fany, Hassig Raymonde, Rey Maria, Dufour Noëlle, Saudou Frédéric, Perrier Anselme L, Hantraye Philippe, Déglon Nicole
Abstract excerpt
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 strategy, a selective silencing of mutant HTT represents the safest approach preserving WT HTT expression and functions. We...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.