基于非病毒载体递送核酸药物的研究进展

Research Progress in the Delivery of Nucleic Acid Drugs Based on Non-Viral Vectors

基于核酸药物的治疗是一种很有前途的疾病治疗策略,通过引入信使RNA(mRNA)、小干扰RNA(siRNA)、微小RNA(miRNA)或反义寡核苷酸(ASO)等外源核酸来调节特定细胞中靶基因表达来治疗疾病。然而,由于它们的负电荷和相当大的尺寸,需要有效地跨膜递送载体选择合适的载体进行递送,将使核酸分子到达其作用位点,提高递送效率。作为有前景的递送系统,非病毒载体由于其低细胞毒性和非免疫原性而受到了广泛的关注。在这篇综述中,详细介绍了非病毒递送系统的最新进展,如基于脂质的纳米颗粒、聚合物纳米颗粒、无机纳米颗粒、外泌体和核酸偶联物。

Nucleic acid-based therapy is a promising strategy for disease treatment,which involves regulating the expression of specific target genes in certain cells by introducing exogenous nucleic acids such as messenger RNA(mRNA),small interfering RNA(siRNA),microRNA(miRNA),or antisense oligonucleotides(ASO).However,due to their negative charge and considerable size,effective transmembrane delivery carriers are required to select suitable carriers for delivery,which can help nucleic acid molecules reach their sites of action and improve delivery efficiency.As a promising delivery system,non-viral carriers have received widespread attention due to their low cytotoxicity and non-immunogenicity.In this review,the latest progress of non-viral delivery systems,such as lipid-based nanoparticles,polymer nanoparticles,inorganic nanoparticles,extracellular vesicles,and nucleic acid conjugates,are described in detail.