摘要/Abstract

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。
关键词: CRISPR-Cas9, 基因编辑, 非肿瘤性血液病, 应用
Abstract:
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-specific 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 symptomscorrecting disease-causing mutations in the near future. Prior to the of CRISPR-Cas9-mediated gene correction in humans, appropriate delivery systems with high efficiency and specificity must be identified, 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.
Key words: CRISPR-Cas9, genome editing, non-cancerous hematological disorder, application


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