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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Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: Public Library of Science 2014
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Research Article
Accesso online: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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