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; Sun, Qiang3; Huang, Xingxu1,6,7
2019-05-31
Source PublicationISCIENCE
ISSN2589-0042
Volume15Pages:640-+
Status已发表
DOI10.1016/j.isci.2019.05.008
Abstract

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.

Indexed BySCI ; ESCI
Funding ProjectNational Postdoctoral Program for Innovative Talents[BX201700266] ; National Postdoctoral Program for Innovative Talents[16JC1420200] ; National Postdoctoral Program for Innovative Talents[17JC1420103]
WOS Research AreaScience & Technology - Other Topics
WOS SubjectMultidisciplinary Sciences
WOS IDWOS:000470104600048
PublisherCELL PRESS
Original Document TypeArticle
Citation statistics
Cited Times:12[WOS]   [WOS Record]     [Related Records in WOS]
Document Type期刊论文
Identifierhttps://kms.shanghaitech.edu.cn/handle/2MSLDSTB/49006
Collection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
Co-First AuthorLiao, Zhaodi; Li, Xiangyang
Corresponding AuthorMa, Xu; Sun, Qiang; Huang, Xingxu
Affiliation1.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
First Author AffilicationSchool of Life Science and Technology
Corresponding Author AffilicationSchool of Life Science and Technology
First Signature AffilicationSchool of Life Science and Technology
Recommended Citation
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-+.
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-+.
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-+.
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