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Article

Mechanism of allele-selective inhibition of huntingtin expression by duplex RNAs that target CAG repeats: function through the RNAi pathway.

Nucleic acids research - 1 Dec 2012

Hu Jiaxin, Liu Jing, Yu Dongbo, Chu Yongjun, Corey David R

Abstract excerpt

Huntington's disease is an incurable neurodegenerative disorder caused by expansion of a CAG trinucleotide repeat within one allele of the huntingtin (HTT) gene. Agents that block expression of mutant HTT and preserve expression of wild-type HTT target the cause of the disease and are an alternative for therapy. We have previously demonstrated that mismatch-containing duplex RNAs complementary to the expanded...

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Source

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Identifiers

PMID: 23042244
DOI: 10.1093/nar/gks907
PMCID: PMC3526262

Genes

HTT
AGO2

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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