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Article

CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington's disease.

The Journal of clinical investigation - 30 Jun 2017

Yang Su, Chang Renbao, Yang Huiming, Zhao Ting, Hong Yan, Kong Ha Eun, Sun Xiaobo, Qin Zhaohui, Jin Peng, Li Shihua, Li Xiao-Jiang

Abstract excerpt

Huntington's disease is a neurodegenerative disorder caused by a polyglutamine repeat in the Huntingtin gene (HTT). Although suppressing the expression of mutant HTT (mHTT) has been explored as a therapeutic strategy to treat Huntington's disease, considerable efforts have gone into developing allele-specific suppression of mHTT expression, given that loss of Htt in mice can lead to embryonic lethality. It...

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Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 28628038
DOI: 10.1172/JCI92087
PMCID: PMC5490741

Genes

HTT

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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