Department of Chemical and Biolmolecular Engineering, University of California, Berkeley, California 94720, USA.
Pharm Res. 2011 Dec;28(12):2966-82. doi: 10.1007/s11095-011-0549-8. Epub 2011 Aug 9.
Viruses continuously evolve to contend with an ever-changing environment that involves transmission between hosts and sometimes species, immune responses, and in some cases therapeutic interventions. Given the high mutation rate of viruses relative to the timescales of host evolution and drug development, novel drug classes that are readily screened and translated to the clinic are needed. RNA interference (RNAi)-a natural mechanism for specific degradation of target RNAs that is conserved from plants to invertebrates and vertebrates-can potentially be harnessed to yield therapies with extensive specificity, ease of design, and broad application. In this review, we discuss basic mechanisms of action and therapeutic applications of RNAi, including design considerations and areas for future development in the field.
病毒不断进化以应对宿主间不断变化的环境，包括传播、免疫反应，有时还包括治疗干预。鉴于病毒的突变率相对较高，而宿主进化和药物开发的时间尺度却较慢，因此需要易于筛选并能迅速转化为临床应用的新型药物类别。RNA 干扰（RNAi）是一种从植物到无脊椎动物和脊椎动物中保守的特定靶标 RNA 降解的天然机制，具有广泛的特异性、易于设计和广泛的应用，具有潜在的应用价值。在这篇综述中，我们讨论了 RNAi 的作用机制和治疗应用，包括设计考虑因素和该领域的未来发展方向。
