RNA干涉在基因治疗中的应用

RNA干涉(RNA interference，RNAi)是一种可以在细胞内抑制特定基因表达的现象.它由双链RNA产生，可以特异性地降解具有相同或者相似序列的RNA（包括mRNA）.它是一种比反义技术更为有效的方法，具有更高的特异性，逐渐成为研究者关注的焦点.人们已经开始探讨使RNA干涉成为一种比反义药物更为有效药物的可能性及其具体的实施方案， 但是也发现它在成为一种安全有效治疗手段之前还有很长的一段路要走.将从RNA干涉的基本理论入手，分析目前这项技术在治疗研究中的应用以及可能遇到的一些问题.

RNAi as a Gene Therapy Approach

RNAi (RNA Interference) has been recently found as an innate cellular process activated when a double-strand RNA (dsRNA) molecule enters the cell causing the degradation of RNAs of identical or closely similar sequences, including mRNAs. It would suppress the expression of specific genes. It proves to be a more efficient tool than the antisence method with more specificity and now it is the focus of researchers allover the world. People are trying to find ways to make RNAi of an efficient clinical method but they also found that it is a long way to go before it becomes successful at last. To make an analysis of the problems and solutions during the process based on the fundamental knowledge that were already known are reviewed.