Developing ABEmax-NG with Precise Targeting and Expanded Editing Scope to Model Pathogenic Splice Site Mutations In Vivo
Huang, Shisheng1,2; Liao, Zhaodi3,4; Li, Xiangyang1,2; Liu, Zhen3; Li, Guanglei1,5; Li, Jianan1,2; Lu, Zongyang1,2; Zhang, Yu1; Li, Xiajun1; Ma, Xu5
2019-05-31
发表期刊ISCIENCE
ISSN2589-0042
卷号15页码:640-648
发表状态已发表
DOI10.1016/j.isci.2019.05.008
摘要

RNA splicing is related to many human diseases; however, lack of efficient genetic approaches to modulate splicing has prevented us from dissecting their functions in human diseases. Recently developed base editors (BEs) offer a new strategy to modulate RNA splicing by converting conservative splice sites, but it is limited by the editing precision and scope. To overcome the limitations of currently available BE-based tools, we combined SpCas9-NG with ABE-max to generate a new BE, ABEmax-NG. We demonstrated that ABEmax-NG performed precise A.T to G.C conversion with an expanded scope, thus covering many more splicing sites. Taking advantage of this tool, we precisely achieved A.T to G.C conversion exactly at the splice sites. We further modeled pathogenic RNA splicing in vitro and in vivo. Taken together, we successfully generated a versatile tool suitable for precise and broad editing at the splice sites.

收录类别SCI ; SCIE
资助项目National Postdoctoral Program for Innovative Talents[BX201700266] ; National Postdoctoral Program for Innovative Talents[16JC1420200] ; National Postdoctoral Program for Innovative Talents[17JC1420103]
WOS研究方向Science & Technology - Other Topics
WOS类目Multidisciplinary Sciences
WOS记录号WOS:000470104600048
出版者CELL PRESS
原始文献类型Article
引用统计
文献类型期刊论文
条目标识符https://kms.shanghaitech.edu.cn/handle/2MSLDSTB/49006
专题生命科学与技术学院_硕士生
生命科学与技术学院_PI研究组_李夏军组
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_博士生
共同第一作者Liao, Zhaodi; Li, Xiangyang
通讯作者Ma, Xu; Sun, Qiang; Huang, Xingxu
作者单位1.ShanghaiTech Univ, Sch Life Sci & Technol, Shanghai 201210, Peoples R China
2.Univ Chinese Acad Sci, Beijing 100049, Peoples R China
3.Chinese Acad Sci, Inst Neurosci, Key Lab Primate Neurobiol, Ctr Excellence Brain Sci & Intelligence Technol, Shanghai 200031, Peoples R China
4.Univ Chinese Acad Sci, Coll Life Sci, Beijing 100049, Peoples R China
5.Natl Res Inst Family Planning, 12 Dahuishi Rd, Beijing 100081, Peoples R China
6.Chinese Acad Sci, Ctr Excellence Mol Cell Sci, Shanghai Inst Biochem & Cell Biol, Shanghai 200031, Peoples R China
7.Univ Chinese Acad Sci, Shanghai 200031, Peoples R China
第一作者单位生命科学与技术学院
通讯作者单位生命科学与技术学院
第一作者的第一单位生命科学与技术学院
推荐引用方式
GB/T 7714
Huang, Shisheng,Liao, Zhaodi,Li, Xiangyang,et al. Developing ABEmax-NG with Precise Targeting and Expanded Editing Scope to Model Pathogenic Splice Site Mutations In Vivo[J]. ISCIENCE,2019,15:640-648.
APA Huang, Shisheng.,Liao, Zhaodi.,Li, Xiangyang.,Liu, Zhen.,Li, Guanglei.,...&Huang, Xingxu.(2019).Developing ABEmax-NG with Precise Targeting and Expanded Editing Scope to Model Pathogenic Splice Site Mutations In Vivo.ISCIENCE,15,640-648.
MLA Huang, Shisheng,et al."Developing ABEmax-NG with Precise Targeting and Expanded Editing Scope to Model Pathogenic Splice Site Mutations In Vivo".ISCIENCE 15(2019):640-648.
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