Article

Bystander base editing interferes with visual function restoration in Leber congenital amaurosis

2024-10-27

Abstract excerpt

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 amaurosis (LCA), a hereditary retinal disorder, as a therapeutic model. We observed that in rd12 of LCA model mice, t...

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Literature Corpus work: 721cac66-ac3f-5879-b584-82959f306562
DOI: 10.1101/2024.10.23.619839

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