反义基因治疗及其在眼科研究中的应用  

Antisense gene therapy and its application in ophthalmology

作　　者：孙洁[1] 谢立信[1] 

机构地区：[1]山东省眼科研究所,青岛266071

出　　处：《中华眼科杂志》2005年第1期87-90,共4页Chinese Journal of Ophthalmology

摘　　要：反义技术是根据碱基互补原理,以序列特异性方式抑制或阻断某一特定基因表达的有效方法,可用于功能基因组学的研究以及疾病的治疗。近20年,反义技术发展迅速,得到了人们越来越多的关注。反义药物有多种,可主要分为3大类反义寡核苷酸、反义RNA与核酶。人们对多种反义药物进行了大量的体外实验及动物实验,证实其在巨细胞病毒性视网膜炎、艾滋病、肿瘤、节段性回肠炎、哮喘等多种疾病的治疗中效果肯定,部分药物已进入了临床试验阶段。随着对反义技术的作用机制、特异性及药理学作用等研究的深入,其应用范围正在不断扩大,在眼科领域的科学研究及临床治疗方面具有广阔的应用前景。本文对反义基因治疗的作用机制、反义药物以及反义技术在眼科研究中的应用进行了阐述。(中华眼科杂志,2005,418790)Antisense technologies, based on the principles of base complementation, are valuable strategies to inhibit or block the expression of a target gene in a sequence-specific manner, and may be used for functional genomics or therapeutic purposes. In the recent twenty years, they have gained more and more attention. And many advances have been made in the studies of antisense technologies. There are many kinds of antisense agents. Among them, three types of antisense strategies can be classified. Firstly, the use of antisense-oligonucleotides; secondly, the expression of antisense RNA; and thirdly, the triggering of RNA cleavage by catalytically active oligonucleotides referred to as ribozymes. Various animal experiments and clinical trials have been performed using different antisense agents. It is proved that antisense strategies can be effective in therapeutic studies of cytomegalovirus-induced retinitis, AIDS, tumors, Crohn′s disease, asthma and so on. Some antisense agents are currently being investigated in Phase Ⅰ-Ⅲ clinical trials. The application of antisense technologies has been expanded greatly with further studies in the mechanism, selection techniques, biostability and pharmacology of antisense agents. Antisense gene therapy shows to be a potential strategy in the experimental study and clinical treatment of ophthalmic diseases. This review introduced the concept, mechanism and application of antisense technologies in ophthalmology.

关 键 词：眼科 反义技术 反义基因治疗 反义药物 肿瘤 作用机制 特异性 科研 科学研究 有效方法 

分 类 号：R77[医药卫生—眼科]