Allele-Selective Suppression of Mutant Huntingtin in Primary Human Blood Cells

Post-transcriptional gene silencing is a promising therapy for the monogenic, autosomal dominant, Huntington’s disease (HD). However, wild-type huntingtin (HTT) has important cellular functions, so the ideal strategy would selectively lower mutant HTT while sparing wild-type. HD patients were genoty...

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Detalhes bibliográficos
Publicado no:Sci Rep
Main Authors: Miller, James R. C., Pfister, Edith L., Liu, Wanzhao, Andre, Ralph, Träger, Ulrike, Kennington, Lori A., Lo, Kimberly, Dijkstra, Sipke, Macdonald, Douglas, Ostroff, Gary, Aronin, Neil, Tabrizi, Sarah J.
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2017
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5402279/
https://ncbi.nlm.nih.gov/pubmed/28436437
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/srep46740
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