Potent and Selective Antisense Oligonucleotides Targeting Single-Nucleotide Polymorphisms in the Huntington Disease Gene / Allele-Specific Silencing of Mutant Huntingtin

Huntington disease (HD) is an autosomal dominant neurodegenerative disorder caused by CAG-expansion in the huntingtin gene (HTT) that results in a toxic gain of function in the mutant huntingtin protein (mHTT). Reducing the expression of mHTT is therefore an attractive therapy for HD. However, wild-...

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Autores principales: Carroll, Jeffrey B, Warby, Simon C, Southwell, Amber L, Doty, Crystal N, Greenlee, Sarah, Skotte, Niels, Hung, Gene, Bennett, C Frank, Freier, Susan M, Hayden, Michael R
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group 2011
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Original Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3242664/
https://ncbi.nlm.nih.gov/pubmed/21971427
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.201
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