摘要
近来随着CRISPR-Cas9精确基因组编辑系统的出现,血液学疾病研究的方法学取得了革命性的进展。CRISPR-Cas9技术被证实可用于去除和纠正基因或突变,并在人类细胞中引入位点特异性治疗基因。因此,该系统介导的基因治疗已经成为遗传性血液疾病治疗的理想方法,并且有望在不久的将来通过纠正致病突变来缓解疾病相关症状。在人类使用CRISPR-Cas9介导的基因校正之前,必须确定具有高效率和特异性的适当的递送系统,同时须建立应用具有可控安全性的技术和道德准则。该文对CRISPR-Cas9技术在非肿瘤性血液学疾病中的应用现状、当前挑战和未来方向进行了综述和讨论。
The recent discovery of the clustered regularly interspaced short palindromic repeats （CRISPR）-CRISPR associated protein 9 （Cas9） system for precise genome editing has revolutionized methodologies in hematology studies. CRISPR-Cas9 technology can remove and correct genes or mutations, and introduce site-specifc therapeutic genes in human cells. Therefore, it has become the ideal targets for inherited haematological disorders and could be the choice to alleviate disease-related symptoms by correcting disease-causing mutations in the near future. Prior to the use of CRISPR-Cas9-mediated gene correction in humans, appropriate delivery systems with high effciency and specifcity must be identifed, and ethical guidelines for applying the technology with controllable safety must be established. In this review, the latest applications of CRISPR-Cas9 technology in haematological disorders, current challenges and future directions were discussed.
作者
翁震
钮晓音
周俊松
WENG Zhen;NIU Xiao-yin;ZHOU Jun-song(Cyrus Tang Hematology Center,Soochow University,Suzhou 215123,China;Shanghai Institute of Immunology,Shanghai Jiao Tong University School of Medicine,Shanghai 200025,China)
出处
《上海交通大学学报（医学版）》
CAS
CSCD
北大核心
2018年第11期1396-1400,共5页
Journal of Shanghai Jiao tong University:Medical Science
基金
国家自然科学基金(81700129
81670133)
上海市卫生和计划生育委员会科研课题(201640137)~~
