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Article

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

American journal of human genetics - 5 Dec 2019

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

Abstract excerpt

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 mutation (A1, A2, A3a) represent panels of allele-specific gene silencing targets for efficient treatment of...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 31708117
DOI: 10.1016/j.ajhg.2019.10.011
PMCID: PMC6904807

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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