Watanabe Tsunamasa, Umehara Takuya, Kohara Michinori
Department of Microbiology and Cell Biology, The Tokyo Metropolitan Institute of Medical Science, 3-18-22, Honkomagome, Bunkyo-ku, Tokyo 113-8613, Japan.
Adv Drug Deliv Rev. 2007 Oct 10;59(12):1263-76. doi: 10.1016/j.addr.2007.03.022. Epub 2007 Aug 11.
RNA interference (RNAi) is a sequence-specific post-transcriptional gene silencing by double-stranded RNA. Because the phenomenon is conserved and ubiquitous in mammalian cells, RNAi has considerable therapeutic potential for human pathogenic gene products. Recent studies have demonstrated the clinical potential of logically designed small interfering RNA (siRNA). However, there are still obstacles in using RNAi as an antiviral therapy, particularly for hepatitis C virus (HCV) that displays a high rate of mutation. Furthermore, delivery is also an important obstacle for siRNA based gene therapy. This paper presents the potential applications and the hurdles facing anti-HCV siRNA drugs. The present review provides insight into the feasible therapeutic strategies of siRNA technology, and its potential for silencing genes associated with HCV disease.
RNA干扰（RNAi）是一种由双链RNA介导的序列特异性转录后基因沉默现象。由于这种现象在哺乳动物细胞中保守且普遍存在，RNAi在治疗人类致病基因产物方面具有巨大的潜力。最近的研究已经证明了合理设计的小干扰RNA（siRNA）的临床潜力。然而，将RNAi用作抗病毒疗法仍存在障碍，尤其是对于具有高突变率的丙型肝炎病毒（HCV）。此外，递送也是基于siRNA的基因治疗的一个重要障碍。本文介绍了抗HCV siRNA药物的潜在应用和面临的障碍。本综述深入探讨了siRNA技术可行的治疗策略及其沉默与HCV疾病相关基因的潜力。
