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Article

Base editing strategies to convert CAG to CAA diminish the disease-causing mutation in Huntington's disease.

eLife - 13 Jun 2024

Choi Doo Eun, Shin Jun Wan, Zeng Sophia, Hong Eun Pyo, Jang Jae-Hyun, Loupe Jacob M, Wheeler Vanessa C, Stutzman Hannah E, Kleinstiver Ben, Lee Jong-Min

Abstract excerpt

An expanded CAG repeat in the huntingtin gene (HTT) causes Huntington's disease (HD). Since the length of uninterrupted CAG repeat, not polyglutamine, determines the age-at-onset in HD, base editing strategies to convert CAG to CAA are anticipated to delay onset by shortening the uninterrupted CAG repeat. Here, we developed base editing strategies to convert CAG in the repeat to CAA and determined their molecular...

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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: 38869243
DOI: 10.7554/eLife.89782
PMCID: PMC11175616

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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