Efficient generation of mouse models of human diseases via ABE- and BE-mediated base editing

Zhen Liu, Zongyang Lu, Guang Yang, Shisheng Huang, Guanglei Li, Songjie Feng, Yajing Liu, Jianan Li, Wenxia Yu, Yu Zhang, Jia Chen, Qiang Sun () and Xingxu Huang ()
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Zhen Liu: Shanghai Institutes for Biological Sciences, CAS
Zongyang Lu: ShanghaiTech University
Guang Yang: ShanghaiTech University
Shisheng Huang: ShanghaiTech University
Guanglei Li: ShanghaiTech University
Songjie Feng: Shanghai Institutes for Biological Sciences, CAS
Yajing Liu: ShanghaiTech University
Jianan Li: ShanghaiTech University
Wenxia Yu: ShanghaiTech University
Yu Zhang: ShanghaiTech University
Jia Chen: ShanghaiTech University
Qiang Sun: Shanghai Institutes for Biological Sciences, CAS
Xingxu Huang: ShanghaiTech University

Nature Communications, 2018, vol. 9, issue 1, 1-8

Abstract: Abstract A recently developed adenine base editor (ABE) efficiently converts A to G and is potentially useful for clinical applications. However, its precision and efficiency in vivo remains to be addressed. Here we achieve A-to-G conversion in vivo at frequencies up to 100% by microinjection of ABE mRNA together with sgRNAs. We then generate mouse models harboring clinically relevant mutations at Ar and Hoxd13, which recapitulates respective clinical defects. Furthermore, we achieve both C-to-T and A-to-G base editing by using a combination of ABE and SaBE3, thus creating mouse model harboring multiple mutations. We also demonstrate the specificity of ABE by deep sequencing and whole-genome sequencing (WGS). Taken together, ABE is highly efficient and precise in vivo, making it feasible to model and potentially cure relevant genetic diseases.

Date: 2018
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DOI: 10.1038/s41467-018-04768-7

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