文献类型：期刊文献

中文题名：基因编辑与诱导多能干细胞的联合应用

英文题名：Cmbination of genome editing and induced pluripotent stem cells

作者：蒋如如[1,2,3];李欣(综述)[1,2,3];哈小琴(审校)[1,2,3]

第一作者：蒋如如

机构：[1]甘肃中医药大学临床医学院,甘肃兰州730030;[2]人民解放军联勤保障部队第九四〇医院检验科,甘肃兰州730050;[3]甘肃省干细胞与基因药物重点实验室,甘肃兰州730050

第一机构：甘肃中医药大学临床医学院

年份：2019

卷号：32

期号：10

起止页码：1143

中文期刊名：中国生物制品学杂志

外文期刊名：Chinese Journal of Biologicals

收录：CSTPCD;;Scopus;CSCD:【CSCD_E2019_2020】；

基金：江苏省科技重大专项(SBA2016002050)

语种：中文

中文关键词：诱导多能干细胞;基因编辑;细胞治疗

外文关键词：Induced pluripotent stem cells(iPSCs);Genome editing;Cell therapy

摘要：基于干细胞的细胞疗法是目前最有潜力和创新性的方法,诱导多能干细胞(induced pluripotent stem cells,iPSC)结合成体干细胞和胚胎干细胞(embryonic stem cell,ESC)的优点和特征,在重编辑技术方面取得了重大进展。本文介绍了iPSC技术和位点特异性核酸酶(site specific nucleotide,SSN)介导的基因编辑工具的发展,以及这两种新技术在生物医学研究和治疗试验中的联合应用。iPSC在体外的多能特性不仅为基础研究提供了无限的细胞来源,而且还有益于人类疾病治疗药物的筛选。此外,随着基因组编辑技术的发展,通过使用核酸酶,如锌指核酸酶(zinc finger nuclease,ZFN)、转录激活因子样效应核酸酶(transcription activator-like effector nucleases,TALEN),以及成簇的、规则间隔短的短回文重复序列(clustered regularly interspaced short palindromic repeats,CRISPR)/Cas系统来纠正基因突变导致的疾病,以便于逆转体细胞突变引起的疾病。将iPSC和SSN技术联合起来创建具有体外同基因背景的新的可靠的人类疾病模型,为精确的细胞替代治疗提供新的解决方案。
Stem-cell-based therapies are considered to be the most promising and innovative approaches.Induced pluripotent stem cells(iPSCs)combine the advantages and unique characteristics of adult stem cells and embryonic stem cells(ESCs).Major progress has already been achieved in reprogramming technology.In this review,we focus on current developments gene editing tools mediated with iPSCs and site-specific nuclease(SSN)and the combined application of these two novel technologies in biomedical research and therapeutic trials.The sustainable pluripotent property of iPSCs in vitro not only provides unlimited cell sources for basic research but also benefits the screening of medicines for human diseases.Besides,genome editing has also been developed further,by including the use of nucleases such as zinc-finger nucleases(ZFN),transcription activator-like effector nucleases(TALEN),clustered regularly interspaced short palindromic repeat/CRISPR-associated system 9(CRISPR/Cas9),to correct the mutagenic diseases and reverse the disease caused by somatic mutation.Combining iPSC and SSN technologies will create new reliable human disease models with isogenic backgrounds in vitro and provide new solutions for precise therapies with cell replacement.