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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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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2014
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4057216/
https://www.ncbi.nlm.nih.gov/pubmed/24926995
http://dx.doi.org/10.1371/journal.pone.0099341