Yang Fei-Fei, Huang Wei, Li Yun-Fei, Gao Zhong-Gao
State Key Laboratory of Bioactive Substances and Functions of Natural Medicines, Institute of Materia Medica, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100050, China.
Yao Xue Xue Bao. 2011 Dec;46(12):1436-43.
RNA interference (RNAi) is a newly developed technology. It is the different levels of gene silencing induced by specific degradation of targeted genes in vivo, and both exogenous and endogenous double-stranded RNAs could induce the specific degradation. RNAi has been applied in tumor therapy, viral infection, hepatitis B and many other diseases. siRNA is the effector molecule which induces the RNAi in vivo. But naked siRNA is easily degradated by RNases in vivo, and the half-life is short. Meanwhile, the transfection efficiency of the naked siRNA is comparatively low. So the naked siRNA needs the help of vectors to penetrate the cell membrane and take action. Viral vectors have the potential immunogenicity and mutagenicity in gene therapy. Therefore, non-viral vectors are drawing more and more attention. The latest development of the non-viral vectors is summarized in this review.
RNA干扰（RNAi）是一项新开发的技术。它是通过体内靶向基因的特异性降解所诱导的不同水平的基因沉默，外源性和内源性双链RNA均可诱导特异性降解。RNAi已应用于肿瘤治疗、病毒感染、乙型肝炎及许多其他疾病。小干扰RNA（siRNA）是体内诱导RNAi的效应分子。但裸露的siRNA在体内易被核糖核酸酶降解，半衰期短。同时，裸露siRNA的转染效率相对较低。因此，裸露的siRNA需要载体的帮助才能穿透细胞膜并发挥作用。病毒载体在基因治疗中具有潜在的免疫原性和致突变性。因此，非病毒载体越来越受到关注。本文综述了非病毒载体的最新进展。
