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Article

Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism.

Chemistry & biology - 24 Nov 2010

Hu Jiaxin, Liu Jing, Corey David R

Abstract excerpt

Inhibiting expression of huntingtin (HTT) protein is a promising strategy for treating Huntington's disease (HD), but indiscriminant inhibition of both wild-type and mutant alleles may lead to toxicity. An ideal silencing agent would block expression of mutant HTT while leaving expression of wild-type HTT intact. We observe that fully complementary duplex RNAs targeting the expanded CAG repeat within HTT mRNA...

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Identifiers

PMID: 21095568
DOI: 10.1016/j.chembiol.2010.10.013
PMCID: PMC3021381

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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Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat.Semantic proximity Read ↗

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