中国组织工程研究2017，Vol.21Issue(9)：1463-1469,7.DOI:10.3969/j.issn.2095-4344.2017.09.027
基因修复β-地中海贫血患者诱导多能干细胞可否成为治疗的希望?
Gene therapy with induced pluripotent stem cells:a hope for beta thalassemia?
摘要
Abstract
BACKGROUND: Beta thalassaemia is a monogenic disease, which lacks effective clinical treatments. Hematopoieticstem cell transplantation currently is the only radical treatment for beta thalassaemia, but the limits of suitable donor and costs minimize its clinical application. Given the technology of reprogramming using somatic cells is well established,gene therapy using induced pluripotent stem cells has become the new direction of beta thalassaemia treatment.OBJECTIVE: To put forward the advantages of CRISPR/Cas9 technology in gene therapy of beta thalassaemia in thefuture by summarizing the mechanisms of three kinds of gene editing technologies and the preliminary experimentalresults in animal models.METHODS: In order to search relevant articles about beta thalassaemia, the first author retrieved PubMed database andCNKI (from 1989 to 2015) using the key words of "beta thalassemia, genetic therapy, genome editing, homologousrecombination, iPSCs" in English and Chinese, respectively. After eliminating literatures which were irrelevant toresearch purpose or containing a similar content, 67 articles were chosen for further analysis.RESULTS AND CONCLUSION: Gene editing technology has made considerable progress and three kinds of directedgene editing technologies have been developed, including ZFNs, TALENs, CRISPR/Cas technology. By targeting inducedpluripotent stem cells from thalassemi patients, these three kinds of gene editing technologies have been expected tocorrect pathogenic genes of thalassemia. The CRISPR/Cas system is more simple, rapid, safe and efficient than the others.The CRISPR/Cas9 system is expected to repair β-globin genes in the induced pluripotent stem cells, germ cells, fertilizedeggs and embryos from beta thalassaemia patients, laying the foundation for future clinical application.关键词
干细胞/移植/基因治疗/β-地中海贫血/CRISPR/Cas9/诱导多能干细胞分类
医药卫生引用本文复制引用
李玲丽,张风波,李崎,马燕琳..基因修复β-地中海贫血患者诱导多能干细胞可否成为治疗的希望?[J].中国组织工程研究,2017,21(9):1463-1469,7.基金项目
国家国际合作重大专项课题(2014DFA30180) （2014DFA30180）
海南省普通高等学校研究生创新科研课题立项(Hys2016-8) the National International Cooperation Project of China, No. 2014DFA30180 （Hys2016-8）
the Innovative Subjects of Postgraduates in Colleges and Universities in Hainan Province, No. Hys2016-8 （）
