Multiplex precise base editing in cynomolgus monkeys
Name
s41467-020-16173-0.pdf
Description
Published version
Size
2.51 MB
Format
Adobe PDF
Checksum (MD5)
8e682d72132f0a8ae1a6ac8ac1f4f9a2
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
Date Issued
2020
Journal
Nature Communications
Publisher
Springer Science and Business Media LLC
Version
Final published version
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.
MIT Department
McGovern Institute for Brain Research at MIT
Terms of Use
Creative Commons Attribution 4.0 International license
Persistent DSpace Link
DOI of Published Version
https://doi.org/10.1038/S41467-020-16173-0
