Article

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

2023-04-28

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 molecul...

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Literature Corpus work: 9def76c5-5bb5-5c2a-9dfc-4f6e79634a7e
DOI: 10.1101/2023.04.28.538700

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