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Multiplex precise base editing in cynomolgus monkeys

Author(s)
Zhang, Wenhui; Aida, Tomomi; del Rosario, Ricardo CH; Wilde, Jonathan J; Ding, Chenhui; Zhang, Xiaohui; Baloch, Zulqurain; Huang, Yan; Tang, Yu; Li, Duanduan; Lu, Hongyu; Zhou, Yang; Jiang, Minqing; Xu, Dongdong; Fang, Zhihao; Zheng, Zhanhong; Huang, Qunshan; Feng, Guoping; Yang, Shihua; ... Show more Show less
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Creative Commons Attribution 4.0 International license https://creativecommons.org/licenses/by/4.0/
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Abstract
© 2020, The Author(s). Common polygenic diseases result from compounded risk contributed by multiple genetic variants, meaning that simultaneous correction or introduction of single nucleotide variants is required for disease modeling and gene therapy. Here, we show precise, efficient, and simultaneous multiplex base editing of up to three target sites across 11 genes/loci in cynomolgus monkey embryos using CRISPR-based cytidine- and adenine-base editors. Unbiased whole genome sequencing demonstrates high specificity of base editing in monkey embryos. Our data demonstrate feasibility of multiplex base editing for polygenic disease modeling in primate zygotes.
Date issued
2020
URI
https://hdl.handle.net/1721.1/133744
Department
McGovern Institute for Brain Research at MIT
Journal
Nature Communications
Publisher
Springer Science and Business Media LLC

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