Bystander editing by adenine base editors impairs vision restoration in a mouse model of Leber congenital amaurosis

Base editors (BEs) have emerged as a powerful tool for gene correction with high activity. However, bystander base editing, a byproduct of BEs, presents challenges for precise editing. Here, we investigated the effects of bystander edits on phenotypic restoration in the context of Leber congenital a...

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Bibliographic Details
Main Authors: Seok-Hoon Lee, Jun Wu, Dongjoon Im, Gue-ho Hwang, You Kyeong Jeong, Hui Jiang, Seok Jae Lee, Dong Hyun Jo, William A. Goddard, III, Jeong Hun Kim, Sangsu Bae
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Molecular Therapy: Methods & Clinical Development
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Online Access:http://www.sciencedirect.com/science/article/pii/S2329050125000567
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