Progress and Prospects of Gene Editing in Pluripotent Stem Cells

doi:10.3390/biomedicines11082168

	Progress and Prospects of Gene Editing in Pluripotent Stem Cells
	Zhang, Zhenwu; Bao, Xinyu; Lin, Chao-Po
	2023-08-01
发表期刊	BIOMEDICINES
EISSN	2227-9059
卷号	11 期号:8
发表状态	已发表
DOI	10.3390/biomedicines11082168
摘要	Applying programmable nucleases in gene editing has greatly shaped current research in basic biology and clinical translation. Gene editing in human pluripotent stem cells (PSCs), including embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs), is highly relevant to clinical cell therapy and thus should be examined with particular caution. First, since all mutations in PSCs will be carried to all their progenies, off-target edits of editors will be amplified. Second, due to the hypersensitivity of PSCs to DNA damage, double-strand breaks (DSBs) made by gene editing could lead to low editing efficiency and the enrichment of cell populations with defective genomic safeguards. In this regard, DSB-independent gene editing tools, such as base editors and prime editors, are favored due to their nature to avoid these consequences. With more understanding of the microbial world, new systems, such as Cas-related nucleases, transposons, and recombinases, are also expanding the toolbox for gene editing. In this review, we discuss current applications of programmable nucleases in PSCs for gene editing, the efforts researchers have made to optimize these systems, as well as new tools that can be potentially employed for differentiation modeling and therapeutic applications.
关键词	pluripotent stem cell induced pluripotent stem cell CRISPR-Cas9 base editor prime editor gene editing
URL	查看原文
收录类别	SCI
语种	英语
WOS研究方向	Biochemistry & Molecular Biology ; Research & Experimental Medicine ; Pharmacology & Pharmacy
WOS类目	Biochemistry & Molecular Biology ; Medicine, Research & Experimental ; Pharmacology & Pharmacy
WOS记录号	WOS:001056653500001
出版者	MDPI
文献类型	期刊论文
条目标识符	https://kms.shanghaitech.edu.cn/handle/2MSLDSTB/327926
专题	生命科学与技术学院生命科学与技术学院_PI研究组_林照博组生命科学与技术学院_硕士生
通讯作者	Lin, Chao-Po
作者单位	ShanghaiTech Univ, Sch Life Sci & Technol, Shanghai 201210, 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	Zhang, Zhenwu,Bao, Xinyu,Lin, Chao-Po. Progress and Prospects of Gene Editing in Pluripotent Stem Cells[J]. BIOMEDICINES,2023,11(8).
APA	Zhang, Zhenwu,Bao, Xinyu,&Lin, Chao-Po.(2023).Progress and Prospects of Gene Editing in Pluripotent Stem Cells.BIOMEDICINES,11(8).
MLA	Zhang, Zhenwu,et al."Progress and Prospects of Gene Editing in Pluripotent Stem Cells".BIOMEDICINES 11.8(2023).