Article
Allele-Selective Suppression of Mutant Huntingtin in Primary Human Blood Cells.
Scientific reports - 24 Apr 2017
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
Abstract excerpt
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 genotyped for heterozygosity at three SNP sites, before phasing each SNP allele to wild-type or mutant HTT. Primary ex...
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