核酶与AIDS治疗

 艾滋病(AIDS)是由人免疫缺陷病毒(HIV)感染并破坏人体免疫功能所导致的一种综合症. 作为具有核酸内切酶活性的RNA小分子，核酶能特异性结合及切割HIV病毒靶分子，并促进靶分子mRNA的裂解，且能相继与多个靶分子RNA作用，同时又不影响宿主细胞RNA. 利用核酶治疗艾滋病不仅没有化疗药物常见的副作用，而且由于核酶本身具有RNA剪切酶活性，可同时剪切HIV mRNA和HIV生命过程中重要的调节蛋白mRNA.因此,较其它基因疗法如RNAi、DNA decoy等,更能有效地抑制HIV复制，并且对由HIV变异而导致的耐药病毒株同样有效，同时对病毒突变的诱导作用也较其它抗病毒药物低.因此,在抗AIDS基因治疗研究中具有潜在的应用价值. 本文在对核酶的结构、催化作用机制及其对HIV-1的作用机制进行概述的基础上，重点讨论了近年来核酶作用于HIV的研究进展以及对AIDS治疗的应用前景.

Ribozymes in AIDS Therapy

AIDS is a kind of syndrome caused by HIV infecting and then destroying human immune system. As a potential gene therapy of AIDS, ribozymes could inhibit the replication of HIV by cutting relative genes of both RNA and regulatory proteins which are important in HIV life cycle; moreover, one ribozyme can cut many target molecules while not destroy the activity of cells. Compared with other gene therapies such as RNAi and DNA decoy, ribozymes could inhibit the replication of HIV more effectively including HIV persisters; on the other hand, the induced mutation rate is lower than other antivirus drugs. The structure, cutting-RNA mechanism and mechanism of action to HIV-1 were introduced in this paper, especially the progress of the studies of the ribozymes acting on HIV in recent years and the foreground in curing AIDS.