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Nat Commun DOI:10.1038/s41467-020-16173-0

Multiplex precise base editing in cynomolgus monkeys.

Publication TypeJournal Article
Year of Publication2020
AuthorsZhang, W, Aida, T, Del Rosario, RCH, Wilde, JJ, Ding, C, Zhang, X, Baloch, Z, Huang, Y, Tang, Y, Li, D, Lu, H, Zhou, Y, Jiang, M, Xu, D, Fang, Z, Zheng, Z, Huang, Q, Feng, G, Yang, S
JournalNat Commun
Volume11
Issue1
Pages2325
Date Published2020 May 11
ISSN2041-1723
Abstract

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.

DOI10.1038/s41467-020-16173-0
Pubmed

http://www.ncbi.nlm.nih.gov/pubmed/32393762?dopt=Abstract

Alternate JournalNat Commun
PubMed ID32393762
PubMed Central IDPMC7214463