Singh Sunit Kumar, Hajeri Praveensingh B
Section of Infectious Diseases & Immunobiology, Centre for Cellular and Molecular Biology, Uppal Road, Hyderabad 500007, AP, India.
Drug Discov Today. 2009 Sep;14(17-18):859-65. doi: 10.1016/j.drudis.2009.06.002. Epub 2009 Jun 21.
RNA interference (RNAi) is a sequence-specific mechanism to control the expression of target genes. This technique has proven potentials both in vivo and in vitro. The main hurdle for using RNAi-based therapy is the effective delivery of RNAi-based drugs to the target cells or tissues in vivo. The aspects of off-target effects, delivery methods, induction of immune response and dose determination for delivery should, however, be considered carefully. If these challenges associated with siRNA can be met, then the potentials of RNAi could be exploited to the full for the development of therapeutic tools and drugs.
RNA干扰（RNAi）是一种控制靶基因表达的序列特异性机制。该技术已在体内和体外均显示出潜力。基于RNAi的疗法的主要障碍是如何在体内将基于RNAi的药物有效递送至靶细胞或组织。然而，脱靶效应、递送方法、免疫反应诱导以及递送剂量的确定等方面都应仔细考虑。如果能够应对与小干扰RNA（siRNA）相关的这些挑战，那么RNAi的潜力就可以被充分利用来开发治疗工具和药物。
