基因工程技术在基因治疗中的应用进展  

Application of genetic engineering on gene therapy

作　　者：刘婷婷(综述) 张殿宝(审校)[2] LIU Ting-ting;ZHANG Dian-bao(Central Hospital of Yingkou Economic and Technological Development Zone(the Sixth People's Hospital of Yingkou),Yingkou 115007,Liaoning,CHINA;College of Basic Medical Sciences,China Medical University,Shenyang 110122,Liaoning,CHINA)

机构地区：[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

出　　处：《海南医学》2021年第4期503-506,共4页Hainan Medical Journal

基　　金：国家自然科学基金(编号:81703102)。

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。Gene therapy was initially used for the treatment of genetic diseases.With the development of genetic engineering technology,it has achieved good clinical effects in the treatment of acquired diseases such as malignant tumors,neurodegenerative diseases,cardiovascular diseases.Several therapeutic strategies have been approved for clinical use.Gene therapy was characterized by high specificity and satisfied therapeutic effects.Gene engineering techniques involved in gene therapy include virus vector technology,gene editing technology and chimeric antigen receptor(CAR)-T technology.Here,the application of gene engineering technology on gene therapy and the research progress of gene therapy are reviewed.

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 

分 类 号：R45[医药卫生—治疗学]