Wu Jian, Nandamuri Kiran Mayi
University of California Davis Medical Center, Department of Internal Medicine, Transplant Research Institute, 4635 2nd Ave, Suite 1001, Sacramento, CA 95817, USA.
Expert Opin Biol Ther. 2004 Oct;4(10):1649-59. doi: 10.1517/14712598.4.10.1649.
Small interfering RNA (siRNA)-mediated sequence-specific gene silencing is a powerful tool to inhibit endogenous and exogenous gene expression, and it holds great potential to prevent and eradicate viral infection, for which existing therapy is inadequate, such as HIV, hepatitis B virus (HBV) and hepatitis C virus (HCV). A number of studies have documented the effectiveness of siRNA against HBV or HCV at various regions of the viral genome in infected human hepatoma cell lines. Selected siRNA may reduce the production of viral replicons, as well as structural or non-structural proteins by > 90%. Only a few in vivo studies that demonstrated the efficacy of siRNA in the suppression of HBV replication in mice are available. Thus, reliable models of HBV and HCV infection in small animals or non-human primates are needed to evaluate the delivery and efficacy of siRNA as a therapeutic modality for viral hepatitis.
小干扰RNA（siRNA）介导的序列特异性基因沉默是抑制内源性和外源性基因表达的有力工具，在预防和根除现有疗法效果不佳的病毒感染（如HIV、乙型肝炎病毒（HBV）和丙型肝炎病毒（HCV））方面具有巨大潜力。许多研究已证明，在受感染的人肝癌细胞系中，针对病毒基因组各个区域的siRNA对HBV或HCV有效。所选的siRNA可使病毒复制子以及结构或非结构蛋白的产生减少90%以上。仅有少数体内研究证明了siRNA在小鼠中抑制HBV复制的功效。因此，需要在小动物或非人类灵长类动物中建立可靠的HBV和HCV感染模型，以评估siRNA作为病毒性肝炎治疗手段的递送效果和疗效。
