关键词: 核酶; RNA; 基因治疗

Abstract: Ribozymes are catalytically active small RNA molecules,which can specifically recognize and bind mRNA of the target gene,and make it fractured and inactivated,thereby blocking or reducing the expression of target genes,and play a role as gene silencing. Besides,ribozymes do not have any adverse effect to cell genomes,so ribozymes as a safe and effective molecular biology tool are increasingly concerned. These properties of ribozymes can be used for gene therapy. For certain pathogens or tumor genes,design specific ribozymes and import them into cells to block or reduce the expression of these genes in cells,ultimately inhibiting pathogen multiplication and tumor proliferation. This article has reviewed the study progress on the treatment of human and animal disease using ribozymes in recent years.

Key words: ribozyme; RNA; gene therapy