RNA干扰(RNA interference,RNAi)是近年发展起来的一种新技术。RNAi是指通过外源性或内源性的双链RNA在体内诱导靶基因mRNA产生特异性降解,进而引起不同水平的基因沉默。RNAi已经用于肿瘤、病毒感染、乙型肝炎等多种疾病的治疗。小干扰RNA(siRNA)是RNAi的效应分子,可在体内诱导RNAi效应。但是裸siRNA在体内容易被核酶(RNase)降解,且半衰期短,转染效率低。因此,siRNA需要借助递送载体进入细胞发挥治疗作用。病毒载体在基因治疗中有潜在的免疫原性、致突变等副作用。所以,非病毒载体成为当前的研究热点。本文对siRNA非病毒递送载体的研究现状进行了综述。
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.