质粒DNA类基因治疗药物的研究现状及发展趋势  

Research status and development trend of plasmid DNA for gene therapy

作　　者：段永平 陈小华 帅明 边原[3] DUAN Yong-ping;CHEN Xiao-hua;SHUAI Ming;BIAN Yuan(College of Chemistry and Life Science,Chifeng University,Chifeng INNER MONGOLIA 024000,China;School of Medicine,University of Electronic Science and Technology of China,Chengdu SICHUAN 610054,China;Department of Pharmacy,Sichuan Academy of Medical Sciences&Sichuan Provincial People's Hospital/Sichuan Provincial Key Laboratory of Personalized Drug Therapy,Affiliated Hospital of University of Electronic Science and Technology of China,Chengdu SICHUAN 610072,China)

机构地区：[1]赤峰学院化学与生命科学学院,内蒙古赤峰024000 [2]电子科技大学医学院,四川成都610054 [3]四川省医学科学院·四川省人民医院、药学部/电子科技大学附属医院、个体化药物治疗四川省重点实验室,四川成都61007

出　　处：《中国新药与临床杂志》2023年第1期8-13,共6页Chinese Journal of New Drugs and Clinical Remedies

基　　金：成都市科技局重点研发支撑计划技术创新研发项目(2020-YF05-00069-SN);四川省科学技术厅2020年省级科技计划(20YYJC1516);国家重点研发计划(2020YFC2005500);四川省科技厅重点研发计划(2019YFS0514);四川省干部保健科研课题(川干研2021-226)。

摘　　要：基因治疗是将外源基因导入人体以纠正基因缺陷的方法,通过转录和翻译实现对细胞基因表达的调控,从而合成特异性蛋白治疗相关疾病。质粒DNA是经基因工程改造过的环状DNA分子,其结构简单,具有自主复制能力,可以携带治疗基因导入人体细胞,被广泛用于基因治疗的研究中。目前已上市及正处于临床研究的基于质粒DNA的基因药物包括cambiogenplasmid、Tavo等。对遗传病、恶性肿瘤等适应证,质粒DNA的基因治疗比传统的治疗方案有明显优势。CRISPR-Cas9等相关的质粒基因编辑技术成为质粒DNA类基因治疗的一大研发趋势,基因枪、聚合物纳米载体等递送系统以透皮给药、静脉注射等方式为质粒DNA导入体内提供了更多可能。基于质粒DNA类的基因治疗已成为基因治疗领域的一种理想技术手段。Gene therapy is a method in which exogenous genes are introduced into the human body to correct gene defects, and the regulation of cellular gene expression is achieved through transcription and translation, thereby synthesizing specific proteins to treat related diseases. Plasmid DNA is a circular DNA molecule modified by genetic engineering. Because of its simple structure and self-replicating ability, it can carry therapeutic genes and then be introduced into human cells. It is widely used in gene therapy research. Plasmid DNA-based gene drugs currently on the market and under clinical research included cambiogenplasmid, Tavo, etc. For the indications such as genetic diseases and malignant tumors, plasmid DNA gene therapy has obvious advantages over traditional therapies. Related plasmid gene editing technologies such as CRISPR-Cas9 have become a major research and development trend in plasmid DNA-based gene therapy. Delivery systems such as gene guns and polymer nanocarriers provide more possibilities for plasmid DNA to be introduced into the body by transdermal administration, intravenous injection and other methods to reach the lesion site to play a therapeutic role. Plasmid DNA-based gene therapy has become an ideal technology in the field of gene therapy.

关 键 词：基因治疗 质粒 DNA 重组 

分 类 号：R394.3[医药卫生—医学遗传学] R915[医药卫生—基础医学]