Chang Ho Lee, Ji Hyun Kim, Seong-Wook Lee, Department of Molecular Biology, Institute of Nanosensor and Biotechnology, Dankook University, Yongin 448-701, South Korea.
World J Gastroenterol. 2013 Dec 21;19(47):8949-62. doi: 10.3748/wjg.v19.i47.8949.
In this review, we discuss recent advances in nucleic acid-based therapeutic technologies that target hepatitis C virus (HCV) infection. Because the HCV genome is present exclusively in RNA form during replication, various nucleic acid-based therapeutic approaches targeting the HCV genome, such as ribozymes, aptamers, siRNAs, and antisense oligonucleotides, have been suggested as potential tools against HCV. Nucleic acids are potentially immunogenic and typically require a delivery tool to be utilized as therapeutics. These limitations have hampered the clinical development of nucleic acid-based therapeutics. However, despite these limitations, nucleic acid-based therapeutics has clinical value due to their great specificity, easy and large-scale synthesis with chemical methods, and pharmaceutical flexibility. Moreover, nucleic acid therapeutics are expected to broaden the range of targetable molecules essential for the HCV replication cycle, and therefore they may prove to be more effective than existing therapeutics, such as interferon-α and ribavirin combination therapy. This review focuses on the current status and future prospects of ribozymes, aptamers, siRNAs, and antisense oligonucleotides as therapeutic reagents against HCV.
在这篇综述中，我们讨论了针对丙型肝炎病毒 (HCV) 感染的基于核酸的治疗技术的最新进展。由于 HCV 基因组在复制过程中仅以 RNA 形式存在，因此针对 HCV 基因组的各种基于核酸的治疗方法，如核酶、适体、siRNA 和反义寡核苷酸，已被提议作为针对 HCV 的潜在工具。核酸具有潜在的免疫原性，通常需要一种输送工具才能用作治疗剂。这些限制阻碍了基于核酸的治疗剂的临床开发。然而，尽管存在这些限制，但基于核酸的治疗剂因其高度特异性、易于通过化学方法进行大规模合成以及药物的灵活性而具有临床价值。此外，核酸治疗剂有望扩大针对 HCV 复制周期的必需靶向分子的范围，因此它们可能比现有的治疗剂（如干扰素-α和利巴韦林联合疗法）更有效。这篇综述重点介绍了核酶、适体、siRNA 和反义寡核苷酸作为针对 HCV 的治疗试剂的现状和未来前景。
