RNA 作为小分子治疗靶点的新兴作用。

Chemical Biology Laboratory, National Cancer Institute, Frederick, MD 21702, USA.

Cell Chem Biol. 2016 Sep 22;23(9):1077-1090. doi: 10.1016/j.chembiol.2016.05.021. Epub 2016 Sep 1.

Recent advances in understanding different RNAs and unique features of their biology have revealed a wealth of information. However, approaches to identify small molecules that target these newly discovered regulatory elements have been lacking. The application of new biochemical screening and design-based technologies, coupled with a resurgence of interest in phenotypic screening, has resulted in several compelling successes in targeting RNA. A number of recent advances suggest that achieving the long-standing goal of developing drug-like, biologically active small molecules that target RNA is possible. This review highlights advances and successes in approaches to targeting RNA with diverse small molecules, and the potential for these technologies to pave the way to new types of RNA-targeted therapeutics.

近年来，人们对不同 RNA 及其生物学独特特征的理解取得了进展，揭示了丰富的信息。然而，针对这些新发现的调控元件的小分子的鉴定方法却一直缺乏。新的生化筛选和基于设计的技术的应用，加上对表型筛选的兴趣再次兴起，使得针对 RNA 的靶向取得了一些令人信服的成功。最近的一些进展表明，实现开发具有生物活性的、类似药物的小分子来靶向 RNA 的长期目标是有可能的。这篇综述强调了利用各种小分子靶向 RNA 的方法的进展和成功，并探讨了这些技术为新型 RNA 靶向治疗方法铺平道路的潜力。