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Article

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

Biochemistry - 30 Nov 2010

Gagnon Keith T, Pendergraff Hannah M, Deleavey Glen F, Swayze Eric E, Potier Pierre, Randolph John, Roesch Eric B, Chattopadhyaya Jyoti, Damha Masad J, Bennett C Frank, Montaillier Christophe, Lemaitre Marc, Corey David R

Abstract excerpt

Huntington's disease (HD) is a currently incurable neurodegenerative disease caused by the expansion of a CAG trinucleotide repeat within the huntingtin (HTT) gene. Therapeutic approaches include selectively inhibiting the expression of the mutated HTT allele while conserving function of the norm...

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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: 21028906
DOI: 10.1021/bi101208k
PMCID: PMC2991413

Genes

HTT

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026. Prepared record publication-21028906-2026-09-02.2. Manifest publication-graph-cohort-2026-09-02.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 2 Sept 2026.

Related research

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Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat.PMID 21028906Read ↗

Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin.Semantic proximity Read ↗

Inhibition of mutant huntingtin expression by RNA duplex targeting expanded CAG repeats.Semantic proximity Read ↗

Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS.Semantic proximity Read ↗

Allele-selective inhibition of mutant huntingtin by peptide nucleic acid-peptide conjugates, locked nucleic acid, and small interfering RNA.Semantic proximity Read ↗

Personalized gene silencing therapeutics for Huntington disease.Semantic proximity Read ↗

Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients.Semantic proximity Read ↗

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

In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides.Semantic proximity Read ↗

Translating Antisense Technology into a Treatment for Huntington's Disease.Semantic proximity Read ↗

Allele-selective inhibition of trinucleotide repeat genes.Semantic proximity Read ↗

Using non-coding small RNAs to develop therapies for Huntington's disease.Semantic proximity Read ↗

CTG repeat-targeting oligonucleotides for down-regulating Huntingtin expression.Semantic proximity Read ↗

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