摘要
RNAi是一种由siRNA触发的特异基因沉默机制。自其发现以来,RNAi技术迅速发展为药物靶标确认和功能基因组学研究的新策略,并有望成为一种疾病基因治疗学方法。然而,由于siRNA体内不稳定的理化性质,递送siRNA成为RNAi发展的最大障碍。因此研发能够保护siRNA、促进细胞吸收、正确细胞定位和包内体逃逸,结合具有高生物相容性与减少非特异沉默和毒性反应的体内递送系统是RNAi成功用于临床治疗的关键。本文就体内非病毒载体方式递送siRNA的研究进展进行综述。
RNAi is a specific gene-silencing mechanism triggered by siRNA.Since its discovery,the RNAi technology has rapidly developed as a new strategy for drug target validation and the study of functional genomics,and it is currently being evaluated as a potential therapy for diseases of a genetic etiology.However,due to the instable physicochemical properties of siRNAs in vivo,the major impediment of RNAi application is the delivery vehicles.Therefore,the research of efficient siRNA delivery for siRNA protection,cellular uptake,correct intracellular localization and endosomal release,combination with high biocompatibility,devoid of nonspecific knockdown and toxic side effects is crucial for the success of RNAi-based therapeutics.In this review,the recent advances of siRNA non-viral vehicles are summarized.
出处
《免疫学杂志》
CAS
CSCD
北大核心
2011年第11期1001-1004,1008,共5页
Immunological Journal
基金
国家自然科学基金(30873027)
