Article
Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients.
Current biology : CB - 12 May 2009
Pfister Edith L, Kennington Lori, Straubhaar Juerg, Wagh Sujata, Liu Wanzhou, DiFiglia Marian, Landwehrmeyer Bernhard, Vonsattel Jean-Paul, Zamore Phillip D, Aronin Neil
Abstract excerpt
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...
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