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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 technology is based on the principle of base complementarity, in a sequence-specific manner, an effective way to inhibit or block the expression of a particular gene can be used for functional genomics research and disease treatment. In the recent 20 years, antisense technology has developed rapidly and attracted more and more attention. Antisense drugs have a variety of can be divided into three major categories of antisense oligonucleotides, antisense RNA and ribozyme. A large number of antisense drugs in vitro experiments and animal experiments confirmed that its cytomegalovirus retinitis, AIDS, cancer, Crohn’s disease, asthma and other diseases in the treatment of certain effects, part of the drug Has entered the clinical trial phase. With the deepening of the research on the mechanism, specificity and pharmacology of antisense technology, its application scope is expanding constantly. It has broad application prospects in the field of ophthalmology research and clinical treatment. This article describes the mechanism of antisense gene therapy, antisense drugs and antisense technology in the application of ophthalmology. (Chinese Journal of Ophthalmology, 2005,418,790)