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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書誌詳細
主要な著者: 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
フォーマット: Artigo
言語:Inglês
出版事項: Public Library of Science 2014
主題:
Research Article
オンライン･アクセス: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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