标题：基因工程技术在基因治疗中的应用进展

      作者：刘婷婷 1 综述 张殿宝 2 审校    1.营口经济技术开发区中心医院(营口市第六人民医院)，辽宁 营口 115007；2.中国医科大学基础医学院，辽宁 沈阳 110122

      卷次： 2021年32卷4期

      【摘要】 基因治疗最初用于治疗遗传性疾病，随着基因工程技术的发展，它在恶性肿瘤、神经退行性疾病、心血管疾病等获得性疾病的治疗中取得良好的临床效果，多种治疗方法获得监管部门的批准上市。基因治疗的优势在于特异性强、靶向性高、效果显著。基因治疗所涉及的基因工程技术包括病毒载体技术、基因编辑技术以及细胞改造的嵌合抗原受体修饰的T细胞技术。本篇综述将探讨基因工程技术在基因治疗中的应用以及基因治疗在国内外的研究进展。
      【关键词】 基因治疗；γ-逆转录病毒载体；慢病毒载体；腺病毒载体；基因编辑；嵌合抗原受体修饰的T细胞
      【中图分类号】 R45 【文献标识码】 A 【文章编号】 1003—6350（2021）04—0503—04

Application of genetic engineering on gene therapy.
LIU Ting-ting 1, ZHANG Dian-bao 2. 1. Central Hospital ofYingkou Economic and Technological Development Zone (the Sixth People's Hospital of Yingkou), Yingkou 115007,Liaoning, CHINA; 2. College of Basic Medical Sciences, China Medical University, Shenyang 110122, Liaoning, CHINA【Abstract】 Gene therapy was initially used for the treatment of genetic diseases. With the development of genet-ic engineering technology, it has achieved good clinical effects in the treatment of acquired diseases such as malignant tu-mors, neurodegenerative diseases, cardiovascular diseases. Several therapeutic strategies have been approved for clinicaluse. Gene therapy was characterized by high specificity and satisfied therapeutic effects. Gene engineering techniques in-volved in gene therapy include virus vector technology, gene editing technology and chimeric antigen receptor (CAR)-Ttechnology. Here, the application of gene engineering technology on gene therapy and the research progress of gene ther-apy are reviewed.
      【Key words】 Gene therapy; γ-retroviral vector; Lentiviral vector; Adeno-associated virus (AAV); Gene editing;Chimeric antigen receptor (CAR) T cells·综述·doi:10.3969/j.issn.1003-6350.2021.04.026基金项目：国家自然科学基金(编号：81703102)