Huntingtin Haplotypes Provide Prioritized Target Panels for Allele-specific Silencing in Huntington Disease Patients of European Ancestry

Huntington disease (HD) is a dominant neurodegenerative disorder caused by a CAG repeat expansion in the Huntingtin gene (HTT). Heterozygous polymorphisms in cis with the mutation allow for allele-specific suppression of the pathogenic HTT transcript as a therapeutic strategy. To prioritize target s...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Kay, Chris, Collins, Jennifer A, Skotte, Niels H, Southwell, Amber L, Warby, Simon C, Caron, Nicholas S, Doty, Crystal N, Nguyen, Betty, Griguoli, Annamaria, Ross, Colin J, Squitieri, Ferdinando, Hayden, Michael R
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2015
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4817952/
https://ncbi.nlm.nih.gov/pubmed/26201449
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.128
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