Efficient generation of mouse models of human diseases via ABE- and BE-mediated base editing
Liu, Zhen1; Lu, Zongyang2; Yang, Guang2; Huang, Shisheng2; Li, Guanglei2; Feng, Songjie1; Liu, Yajing2; Li, Jianan2; Yu, Wenxia2; Zhang, Yu2; Chen, Jia2; Sun, Qiang1; Huang, Xingxu2,3
2018-06-14
Source PublicationNATURE COMMUNICATIONS
ISSN2041-1723
Volume9
Status已发表
DOI10.1038/s41467-018-04768-7
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.

Indexed BySCI
Funding Project[17JC1420103]
WOS Research AreaScience & Technology - Other Topics
WOS SubjectMultidisciplinary Sciences
WOS IDWOS:000435341000002
PublisherNATURE PUBLISHING GROUP
WOS KeywordDNA CLEAVAGE ; GENOMIC DNA ; MUTATIONS
Original Document TypeArticle
Citation statistics
Cited Times:73[WOS]   [WOS Record]     [Related Records in WOS]
Document Type期刊论文
Identifierhttps://kms.shanghaitech.edu.cn/handle/2MSLDSTB/27445
Collection生命科学与技术学院
生命科学与技术学院_PI研究组_陈佳组
生命科学与技术学院_PI研究组_黄行许组
生命科学与技术学院_硕士生
生命科学与技术学院_博士生
Co-First AuthorLu, Zongyang; Yang, Guang
Corresponding AuthorSun, Qiang; Huang, Xingxu
Affiliation1.Chinese Acad Sci, Inst Neurosci, Key Lab Primate Neurobiol,Shanghai Inst Biol Sci, CAS Ctr Excellence Brain Sci & Intelligence Techn, Shanghai 200031, Peoples R China
2.ShanghaiTech Univ, Sch Life Sci & Technol, 100 Haike Rd, Shanghai 201210, Peoples R China
3.Univ Chinese Acad Sci, Chinese Acad Sci, Shanghai Inst Biochem & Cell Biol, CAS Ctr Excellence Mol Cell Sci, 320 Yueyang Rd, Shanghai 200031, Peoples R China
Corresponding Author AffilicationSchool of Life Science and Technology
Recommended Citation
GB/T 7714
Liu, Zhen,Lu, Zongyang,Yang, Guang,et al. Efficient generation of mouse models of human diseases via ABE- and BE-mediated base editing[J]. NATURE COMMUNICATIONS,2018,9.
APA Liu, Zhen.,Lu, Zongyang.,Yang, Guang.,Huang, Shisheng.,Li, Guanglei.,...&Huang, Xingxu.(2018).Efficient generation of mouse models of human diseases via ABE- and BE-mediated base editing.NATURE COMMUNICATIONS,9.
MLA Liu, Zhen,et al."Efficient generation of mouse models of human diseases via ABE- and BE-mediated base editing".NATURE COMMUNICATIONS 9(2018).
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