siRNA药物的临床研究进展

摘要

RNA干扰技术作为一种新型的基因沉默技术,因其具有高效、高特异性、低毒性等优点,应用RNA干扰技术开发新型的靶向药物,已成为当今药物研究领域中重点发展方向之一.siRNA作为药物应用于临床已经取得很大的发展但仍面临许多挑战.本文对RNA干扰机制、临床试验现状及发展前景作简要综述.%The ability to manipulate the RNA interference (RNAi) machinery to specifically silence the expression of targets could be a powerful therapeutic strategy. There has been a tremendous push by researchers to move siRNAs into clinical application. However, the challenges facing siRNA therapeutics are significant. This review presented the clinical application and comments on broad prospects for the approach.