Cytosine base editors with increased PAM and deaminase motif flexibility for gene editing in zebrafish

Abstract Cytosine base editing is a powerful tool for making precise single nucleotide changes in cells and model organisms like zebrafish, which are valuable for studying human diseases. However, current base editors struggle to edit cytosines in certain DNA contexts, particularly those with GC and...

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Main Authors: Yu Zhang, Yang Liu, Wei Qin, Shaohui Zheng, Jiawang Xiao, Xinxin Xia, Xuanyao Yuan, Jingjing Zeng, Yu Shi, Yan Zhang, Hui Ma, Gaurav K. Varshney, Ji-Feng Fei, Yanmei Liu
Format: Article
Language:English
Published: Nature Portfolio 2024-11-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-024-53735-y
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author Yu Zhang
Yang Liu
Wei Qin
Shaohui Zheng
Jiawang Xiao
Xinxin Xia
Xuanyao Yuan
Jingjing Zeng
Yu Shi
Yan Zhang
Hui Ma
Gaurav K. Varshney
Ji-Feng Fei
Yanmei Liu
author_facet Yu Zhang
Yang Liu
Wei Qin
Shaohui Zheng
Jiawang Xiao
Xinxin Xia
Xuanyao Yuan
Jingjing Zeng
Yu Shi
Yan Zhang
Hui Ma
Gaurav K. Varshney
Ji-Feng Fei
Yanmei Liu
author_sort Yu Zhang
collection DOAJ
description Abstract Cytosine base editing is a powerful tool for making precise single nucleotide changes in cells and model organisms like zebrafish, which are valuable for studying human diseases. However, current base editors struggle to edit cytosines in certain DNA contexts, particularly those with GC and CC pairs, limiting their use in modelling disease-related mutations. Here we show the development of zevoCDA1, an optimized cytosine base editor for zebrafish that improves editing efficiency across various DNA contexts and reduces restrictions imposed by the protospacer adjacent motif. We also create zevoCDA1-198, a more precise editor with a narrower editing window of five nucleotides, minimizing off-target effects. Using these advanced tools, we successfully generate zebrafish models of diseases that were previously challenging to create due to sequence limitations. This work enhances the ability to introduce human pathogenic mutations in zebrafish, broadening the scope for genomic research with improved precision and efficiency.
format Article
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institution DOAJ
issn 2041-1723
language English
publishDate 2024-11-01
publisher Nature Portfolio
record_format Article
series Nature Communications
spelling doaj-art-6a182ad0fa8c4a7598f100335ea9385a2025-08-20T02:50:04ZengNature PortfolioNature Communications2041-17232024-11-0115111010.1038/s41467-024-53735-yCytosine base editors with increased PAM and deaminase motif flexibility for gene editing in zebrafishYu Zhang0Yang Liu1Wei Qin2Shaohui Zheng3Jiawang Xiao4Xinxin Xia5Xuanyao Yuan6Jingjing Zeng7Yu Shi8Yan Zhang9Hui Ma10Gaurav K. Varshney11Ji-Feng Fei12Yanmei Liu13Key Laboratory of Brain, Cognition and Education Sciences, Ministry of Education, South China Normal UniversityKey Laboratory of Brain, Cognition and Education Sciences, Ministry of Education, South China Normal UniversityGenes & Human Disease Research Program, Oklahoma Medical Research FoundationKey Laboratory of Brain, Cognition and Education Sciences, Ministry of Education, South China Normal UniversityKey Laboratory of Brain, Cognition and Education Sciences, Ministry of Education, South China Normal UniversityKey Laboratory of Brain, Cognition and Education Sciences, Ministry of Education, South China Normal UniversityKey Laboratory of Brain, Cognition and Education Sciences, Ministry of Education, South China Normal UniversityKey Laboratory of Brain, Cognition and Education Sciences, Ministry of Education, South China Normal UniversityKey Laboratory of Brain, Cognition and Education Sciences, Ministry of Education, South China Normal UniversityKey Laboratory of Brain, Cognition and Education Sciences, Ministry of Education, South China Normal UniversityDepartment of Pathology, Guangdong Provincial People’s Hospital (Guangdong Academy of Medical Sciences), Southern Medical UniversityGenes & Human Disease Research Program, Oklahoma Medical Research FoundationDepartment of Pathology, Guangdong Provincial People’s Hospital (Guangdong Academy of Medical Sciences), Southern Medical UniversityKey Laboratory of Brain, Cognition and Education Sciences, Ministry of Education, South China Normal UniversityAbstract Cytosine base editing is a powerful tool for making precise single nucleotide changes in cells and model organisms like zebrafish, which are valuable for studying human diseases. However, current base editors struggle to edit cytosines in certain DNA contexts, particularly those with GC and CC pairs, limiting their use in modelling disease-related mutations. Here we show the development of zevoCDA1, an optimized cytosine base editor for zebrafish that improves editing efficiency across various DNA contexts and reduces restrictions imposed by the protospacer adjacent motif. We also create zevoCDA1-198, a more precise editor with a narrower editing window of five nucleotides, minimizing off-target effects. Using these advanced tools, we successfully generate zebrafish models of diseases that were previously challenging to create due to sequence limitations. This work enhances the ability to introduce human pathogenic mutations in zebrafish, broadening the scope for genomic research with improved precision and efficiency.https://doi.org/10.1038/s41467-024-53735-y
spellingShingle Yu Zhang
Yang Liu
Wei Qin
Shaohui Zheng
Jiawang Xiao
Xinxin Xia
Xuanyao Yuan
Jingjing Zeng
Yu Shi
Yan Zhang
Hui Ma
Gaurav K. Varshney
Ji-Feng Fei
Yanmei Liu
Cytosine base editors with increased PAM and deaminase motif flexibility for gene editing in zebrafish
Nature Communications
title Cytosine base editors with increased PAM and deaminase motif flexibility for gene editing in zebrafish
title_full Cytosine base editors with increased PAM and deaminase motif flexibility for gene editing in zebrafish
title_fullStr Cytosine base editors with increased PAM and deaminase motif flexibility for gene editing in zebrafish
title_full_unstemmed Cytosine base editors with increased PAM and deaminase motif flexibility for gene editing in zebrafish
title_short Cytosine base editors with increased PAM and deaminase motif flexibility for gene editing in zebrafish
title_sort cytosine base editors with increased pam and deaminase motif flexibility for gene editing in zebrafish
url https://doi.org/10.1038/s41467-024-53735-y
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