Feng Qiang, Li Qirong, Zhou Hengzong, Wang Zhan, Lin Chao, Jiang Ziping, Liu Tianjia, Wang Dongxu
Laboratory Animal Center College of Animal Science Jilin University Changchun China.
Research and Development Centre Baicheng Medical College Baicheng China.
MedComm (2020). 2024 Jul 29;5(8):e672. doi: 10.1002/mco2.672. eCollection 2024 Aug.
Gene editing is a growing gene engineering technique that allows accurate editing of a broad spectrum of gene-regulated diseases to achieve curative treatment and also has the potential to be used as an adjunct to the conventional treatment of diseases. Gene editing technology, mainly based on clustered regularly interspaced palindromic repeats (CRISPR)-CRISPR-associated protein systems, which is capable of generating genetic modifications in somatic cells, provides a promising new strategy for gene therapy for a wide range of human diseases. Currently, gene editing technology shows great application prospects in a variety of human diseases, not only in therapeutic potential but also in the construction of animal models of human diseases. This paper describes the application of gene editing technology in hematological diseases, solid tumors, immune disorders, ophthalmological diseases, and metabolic diseases; focuses on the therapeutic strategies of gene editing technology in sickle cell disease; provides an overview of the role of gene editing technology in the construction of animal models of human diseases; and discusses the limitations of gene editing technology in the treatment of diseases, which is intended to provide an important reference for the applications of gene editing technology in the human disease.
基因编辑是一种不断发展的基因工程技术，它能够对多种基因调控疾病进行精确编辑以实现治愈性治疗，并且还有潜力用作疾病传统治疗的辅助手段。基因编辑技术主要基于成簇规律间隔短回文重复序列（CRISPR）-CRISPR相关蛋白系统，该系统能够在体细胞中产生基因修饰，为多种人类疾病的基因治疗提供了一种有前景的新策略。目前，基因编辑技术在多种人类疾病中展现出巨大的应用前景，不仅体现在治疗潜力方面，还体现在人类疾病动物模型的构建上。本文介绍了基因编辑技术在血液系统疾病、实体瘤、免疫紊乱、眼科疾病和代谢疾病中的应用；重点阐述了基因编辑技术在镰状细胞病中的治疗策略；概述了基因编辑技术在人类疾病动物模型构建中的作用；并讨论了基因编辑技术在疾病治疗中的局限性，旨在为基因编辑技术在人类疾病中的应用提供重要参考。
