A Comprehensive Haplotype-Targeting Strategy for Allele-Specific HTT Suppression in Huntington Disease

Huntington disease (HD) is a fatal neurodegenerative disorder caused by a gain-of-function mutation in HTT. Suppression of mutant HTT has emerged as a leading therapeutic strategy for HD, with allele-selective approaches targeting HTT SNPs now in clinical trials. Haplotypes associated with the HD mu...

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Udgivet i:Am J Hum Genet
Main Authors: Kay, Chris, Collins, Jennifer A., Caron, Nicholas S., Agostinho, Luciana de Andrade, Findlay-Black, Hailey, Casal, Lorenzo, Sumathipala, Dulika, Dissanayake, Vajira H.W., Cornejo-Olivas, Mario, Baine, Fiona, Krause, Amanda, Greenberg, Jacquie L., Paiva, Carmen Lúcia Antão, Squitieri, Ferdinando, Hayden, Michael R.
Format: Artigo
Sprog:Inglês
Udgivet: Elsevier 2019
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6904807/
https://ncbi.nlm.nih.gov/pubmed/31708117
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ajhg.2019.10.011
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