Multiplex precise base editing in cynomolgus monkeys.

Nat Commun
Authors
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.

Year of Publication
2020
Journal
Nat Commun
Volume
11
Issue
1
Pages
2325
Date Published
2020 May 11
ISSN
2041-1723
DOI
10.1038/s41467-020-16173-0
PubMed ID
32393762
PubMed Central ID
PMC7214463
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