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基因治疗的递送系统

Delivery systems for gene therapy.

作者信息

Mali Shrikant

机构信息

Department of Oral and Maxillofacial Surgery, CSMSS Dental College, Aurangabad, Maharashtra, India.

出版信息

Indian J Hum Genet. 2013 Jan;19(1):3-8. doi: 10.4103/0971-6866.112870.

Abstract

The structure of DNA was unraveled by Watson and Crick in 1953, and two decades later Arber, Nathans and Smith discovered DNA restriction enzymes, which led to the rapid growth in the field of recombinant DNA technology. From expressing cloned genes in bacteria to expressing foreign DNA in transgenic animals, DNA is now slated to be used as a therapeutic agent to replace defective genes in patients suffering from genetic disorders or to kill tumor cells in cancer patients. Gene therapy provides modern medicine with new perspectives that were unthinkable two decades ago. Progress in molecular biology and especially, molecular medicine is now changing the basics of clinical medicine. A variety of viral and non-viral possibilities are available for basic and clinical research. This review summarizes the delivery routes and methods for gene transfer used in gene therapy.

摘要

1953年,沃森和克里克解开了DNA的结构之谜。二十年后,阿伯、内森斯和史密斯发现了DNA限制酶,这使得重组DNA技术领域迅速发展。从在细菌中表达克隆基因到在转基因动物中表达外源DNA,如今DNA有望被用作治疗剂,以替代患有遗传疾病患者体内的缺陷基因,或杀死癌症患者体内的肿瘤细胞。基因治疗为现代医学提供了二十年前难以想象的新视角。分子生物学尤其是分子医学的进展正在改变临床医学的基础。基础研究和临床研究有多种病毒和非病毒的方法可供选择。本文综述了基因治疗中使用的基因传递途径和方法。

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