Advances in CRISPR/Cas gene therapy for inborn errors of immunity

doi:10.3389/fimmu.2023.1111777

	Advances in CRISPR/Cas gene therapy for inborn errors of immunity
	Liu, Xinyi 1; Li, Guanglei2 ; Liu, Yin 3; Zhou, Fuling 3; Huang, Xingxu2 ; Li, Kui 1
	2023-03-27
发表期刊	FRONTIERS IN IMMUNOLOGY (IF:5.7[JCR-2023],6.8[5-Year])
ISSN	1664-3224
卷号	14
发表状态	已发表
DOI	10.3389/fimmu.2023.1111777
摘要	Inborn errors of immunity (IEIs) are a group of inherited disorders caused by mutations in the protein-coding genes involved in innate and/or adaptive immunity. Hematopoietic stem cell transplantation (HSCT) is a mainstay definitive therapy for many severe IEIs. However, the lack of HLA-matched donors increases the risk of developing severe immunological complications. Gene therapy provides long-term clinical benefits and could be an attractive therapeutic strategy for IEIs. In this review, we describe the development and evolution of clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated proteins (Cas) gene-editing systems, including double-strand break (DSB)-based gene editing and DSB-free base editing or prime editing systems. Here, we discuss the advances in and issues associated with CRISPR/Cas gene editing tools and their potential as therapeutic alternatives for IEIs. We also highlight the progress of preclinical studies for the treatment of human genetic diseases, including IEIs, using CRISR/Cas and ongoing clinical trials based on this versatile technology.
关键词	inborn errors of immunity (IEIs) Primary Immunodeficiencies (PIDs) CRISPR Cas gene therapy gene editing base editing prime editing
URL	查看原文
收录类别	SCI
语种	英语
资助项目	National Key R&D Program of China[2021YFF100604] ; National Natural Science Foundation of China[32202647]
WOS研究方向	Immunology
WOS类目	Immunology
WOS记录号	WOS:000964953100001
出版者	FRONTIERS MEDIA SA
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/296035
专题	生命科学与技术学院生命科学与技术学院_PI研究组_池天组生命科学与技术学院_PI研究组_黄行许组
通讯作者	Huang, Xingxu; Li, Kui
作者单位	1.Chinese Acad Agr Sci, Agr Genom Inst Shenzhen, Shenzhen Branch, Guangdong Lab Lingnan Modern Agr, Shenzhen, Peoples R China 2.ShanghaiTech Univ, Sch Life Sci & Technol, Shanghai, Peoples R China 3.Wuhan Univ, Zhongnan Hosp, Dept Hematol, Wuhan, Peoples R China
通讯作者单位	生命科学与技术学院
推荐引用方式 GB/T 7714	Liu, Xinyi,Li, Guanglei,Liu, Yin,et al. Advances in CRISPR/Cas gene therapy for inborn errors of immunity[J]. FRONTIERS IN IMMUNOLOGY,2023,14.
APA	Liu, Xinyi,Li, Guanglei,Liu, Yin,Zhou, Fuling,Huang, Xingxu,&Li, Kui.(2023).Advances in CRISPR/Cas gene therapy for inborn errors of immunity.FRONTIERS IN IMMUNOLOGY,14.
MLA	Liu, Xinyi,et al."Advances in CRISPR/Cas gene therapy for inborn errors of immunity".FRONTIERS IN IMMUNOLOGY 14(2023).