Developing ABEmax-NG with Precise Targeting and Expanded Editing Scope to Model Pathogenic Splice Site Mutations In Vivo

doi:10.1016/j.isci.2019.05.008

	Developing ABEmax-NG with Precise Targeting and Expanded Editing Scope to Model Pathogenic Splice Site Mutations In Vivo
	Huang, Shisheng1,2 ; Liao, Zhaodi 3,4; Li, Xiangyang1,2 ; Liu, Zhen 3; Li, Guanglei1,5 ; Li, Jianan1,2 ; Lu, Zongyang1,2 ; Zhang, Yu1 ; Li, Xiajun1 ; Ma, Xu 5; Sun, Qiang 3; Huang, Xingxu1,6,7
	2019-05-31
发表期刊	ISCIENCE (IF:4.6[JCR-2023],5.0[5-Year])
ISSN	2589-0042
卷号	15 页码:640-648
发表状态	已发表
DOI	10.1016/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	正在获取...
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.