Progress in RNA interference therapy.docVIP

 PAGE \* MERGEFORMAT 7 Progress in RNA interference therapy [Keywords:] RNA, interference treatment RNA interference in mammalian cells, gene silencing effect only in recent years found that the current study of gene function has become a tool [1]. Based on RNA interference technology has become the modern study of drug targeting and effectiveness of basic research tools . Because RNA can simulate effects of drugs against, siRNA can be directly developed into its own drugs for the treatment of human diseases, as long as the choice of a good disease target sites, you can bypass the complex and long-term drug development process, thus saving a lot of funding. Therefore, RNAi interference treatment prospects. RNA interference or RNAi-mediated gene silencing by the siRNA, mainly through the degradation of their cognate mRNAs, and this effect has always been throughout the eukaryotic cell. RNAi biotechnology to benefit from the rapid development of foreign gene now known to RNAi into mammalian cells retained the main way is to use miRNA evolution. In mammalian cells, endogenous miRNA gene transcripts produced by Drosha miRNA first body, the beginning of the body in the nucleus and further form miRNA precursor miRNA, miRNA precursors and the formation of the mature miRNA by Exportin5 , miRNA RISC complex integration of form, by inhibiting the degradation of RNA translation or RNA complement complement to regulate gene expression. miRNA has an important physiological role, especially in the early development of malignant tumors. 1 RNAi drug development potential With the traditional small molecule drugs and antibodies would like to compare, RNAi treatment of the following advantages: (1 because it can through the target gene sequence of rational design of drug, from target to drug process was greatly reduced. (2RNAi drug synthesis route is basically the same. ( 3RNAi able to lock any gene therapy, including non-drug and non-protein gene. (4RNAi drugs