School of Biomedical Sciences, Faculty of Medicine, The Chinese University of Hong Kong, Lo Kwee-Seong Integrated Biomedical Sciences Building, Area 39, Sha Tin, New Territories, Hong Kong, China.
Department of Medicine and Therapeutics, Faculty of Medicine, The Chinese University of Hong Kong, Sha Tin, New Territories, Hong Kong, China.
Appl Microbiol Biotechnol. 2017 Oct;101(19):7091-7111. doi: 10.1007/s00253-017-8433-z. Epub 2017 Aug 8.
Short interfering RNA (siRNA) is one of the members of the family of RNA interference (RNAi). Coupled with the RNA-induced silencing complex (RISC), siRNA is able to trigger the cleavage of target RNAs which serve as a defensive system against pathogens. Meanwhile, siRNA in gene silencing opens a new avenue for the treatment of various diseases. SiRNA can effectively inhibit viral infection and replication and suppress tumorigenesis and various inflammation-associated diseases and cardiovascular diseases by inactivation of viral genes and downregulation of oncogene expression. Recently, endogenous siRNAs (endo-siRNAs) were discovered in the reproductive cells of animals which may be associated with regulation of cell division. Structural modification of siRNA enhances the delivery, specificity and efficacy and bioavailability to the target cells. There are at least five categories of siRNA delivery systems including viral vectors, lipid-based nanoparticles, peptide-based nanoparticles, polymer-based nanoparticles and inorganic small molecules like metal ions, silica and carbon. Sufficient preclinical and clinical studies supported that siRNA may be a potential medicine for targeted therapy of various diseases in the near future.
短干扰 RNA（siRNA）是 RNA 干扰（RNAi）家族的成员之一。与 RNA 诱导沉默复合物（RISC）结合后，siRNA 能够触发靶 RNA 的切割，这是一种针对病原体的防御系统。同时，siRNA 在基因沉默中为治疗各种疾病开辟了新途径。siRNA 可以通过病毒基因失活和下调致癌基因表达，有效抑制病毒感染和复制，并抑制肿瘤发生和各种与炎症相关的疾病和心血管疾病。最近，在动物的生殖细胞中发现了内源性 siRNA（endo-siRNA），这可能与细胞分裂的调节有关。siRNA 的结构修饰可增强对靶细胞的递送、特异性和功效以及生物利用度。siRNA 的递送系统至少有五类，包括病毒载体、基于脂质的纳米颗粒、基于肽的纳米颗粒、基于聚合物的纳米颗粒和无机小分子，如金属离子、硅和碳。充足的临床前和临床研究表明，siRNA 可能在不久的将来成为针对各种疾病的靶向治疗的潜在药物。
