Tang Yu, Ge Yu-Zhi, Yin James Q
Institute of Biophysics, Chinese Academy of Sciences, Beijing 100101, China.
Acta Pharmacol Sin. 2007 Jan;28(1):1-9. doi: 10.1111/j.1745-7254.2007.00491.x.
RNA interference (RNAi) is an adaptive defense mechanism through which double stranded RNAs silence cognate genes in a sequence-specific manner. It has been employed widely as a powerful tool in functional genomics studies, target validation and therapeutic product development. Similarly, the application of small interfering RNA (siRNA) to the silencing of the disease-causing genes involved in cardiovascular diseases has made great progress. In this overview, we attempt to provide a brief outline of the current understanding of the mechanism of RNAi and its potential application to the cardiovascular system, with particular emphasis on its ability to identify the pathophysiological function of genes related to several important cardiovascular disorders. The prospects of RNAi-based therapeutics, as well as the advantages and potential problems, are also discussed.
RNA干扰（RNAi）是一种适应性防御机制，双链RNA通过该机制以序列特异性方式使同源基因沉默。它已被广泛用作功能基因组学研究、靶点验证和治疗产品开发的强大工具。同样，小干扰RNA（siRNA）在沉默心血管疾病相关致病基因方面的应用也取得了很大进展。在本综述中，我们试图简要概述目前对RNAi机制的理解及其在心血管系统中的潜在应用，特别强调其识别与几种重要心血管疾病相关基因的病理生理功能的能力。还讨论了基于RNAi的治疗方法的前景以及其优势和潜在问题。
