RNA干扰非特异性研究进展

摘要

RNA干扰(RNAi)靶向基因治疗是目前研究的热点,其依赖序列特异性的基因表达调节为生物学发展带来重大突破.然而在应用RNAi时所产生的一系列非特异性反应阻碍了RNAi进一步的临床应用,这其中主要包括脱靶基因抑制和内在免疫副反应等.主要从非特异性反应的产生机制、脱靶效应的预测和RNAi非特异性的控制3个方面对近几年研究成果进行综述,探讨临床应用前景.%At present, RNA interference is being the research focus for gene therapeutic applications. Despite the excitement about this remarkable biological process for sequence specific gene regulation, there are a number of problems that need to be overcome prior to making RNAi as real therapeutic modality, which can include off-target effects and activating innate immune responses. This review discussed mechanistic aspects, precaution and control of off-target effects and gave a prospect for clinical application.