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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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Bibliografiske detaljer
Main Authors: 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
Format: Artigo
Sprog:Inglês
Udgivet: Public Library of Science 2014
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4057216/
https://ncbi.nlm.nih.gov/pubmed/24926995
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0099341
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