Five siRNAs targeting three SNPs in Huntingtin may provide therapy for three-quarters of Huntington’s disease patients

Among dominant neurodegenerative disorders, Huntington’s disease (HD) is perhaps the best candidate for treatment with small interfering RNAs (siRNAs) [1–9]. Invariably fatal, HD is caused by expansion of a CAG repeat in the Huntingtin gene, creating an extended polyglutamine tract that makes the Hu...

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Main Authors: Pfister, Edith L., Kennington, Lori, Straubhaar, Juerg, Wagh, Sujata, Liu, Wanzhou, DiFiglia, Marian, Landwehrmeyer, Bernhard, Vonsattel, Jean-Paul, Zamore, Phillip D., Aronin, Neil
Format: Artigo
Sprog:Inglês
Udgivet: 2009
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2746439/
https://ncbi.nlm.nih.gov/pubmed/19361997
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.cub.2009.03.030
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