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

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 r...

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Autors principals:	Doo Eun Choi, Jun Wan Shin, Sophia Zeng, Eun Pyo Hong, Jae-Hyun Jang, Jacob M Loupe, Vanessa C Wheeler, Hannah E Stutzman, Ben Kleinstiver, Jong-Min Lee
Format:	Artigo
Idioma:	Inglês
Publicat:	eLife Sciences Publications Ltd 2024-06-01
Col·lecció:	eLife
Matèries:	Huntington's disease duplicated interruption uninterrupted repeat base editing CAG-to-CAA conversion CAG repeat instability
Accés en línia:	https://elifesciences.org/articles/89782
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