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Article

Correcting a patient-specific Rhodopsin mutation with adenine base editor in a mouse model.

Molecular therapy : the journal of the American Society of Gene Therapy - 2 Jul 2025

He Xiaoxue, Yan Tong, Song Zongming, Xiang Lue, Xiang Jiayang, Yang Yeqin, Ren Kaiqun, Bu Jicheng, Xu Xilin, Li Zhuo, Guo Xiaowei, Lin Bin, Zhou Qinghua, Lin Ge, Gu Feng

Abstract excerpt

Genome editing offers a great promise to treating human genetic diseases. To assess genome-editing-mediated therapeutic effects in vivo, an animal model is indispensable. The genomic disparities between mice and humans often impede the direct clinical application of genome-editing-mediated treatments using conventional mouse models. Thus, the generation of a mouse model with a humanized genomic segment containing...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 40119518
DOI: 10.1016/j.ymthe.2025.03.021
PMCID: PMC12265961

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