Small molecule drugs have readily been developed against many proteins in the human proteome, but RNA has remained an elusive target for drug discovery. Increasingly, we see that RNA, and to a lesser extent DNA elements, show a persistent tertiary structure responsible for many diverse and complex cellular functions. In this digest, we have summarized recent advances in screening approaches for RNA targets and outlined the discovery of novel, drug-like small molecules against RNA targets from various classes and therapeutic areas. The link of structure, function, and small-molecule Druggability validates now for the first time that RNA can be the targets of therapeutic agents.

人们已经很容易开发出针对人类蛋白质组中许多蛋白质的小分子药物，但是RNA仍然是药物发现的目标。我们越来越多地看到，RNA和较小程度的DNA元素显示出持久的三级结构，负责许多不同而复杂的细胞功能。在本摘要中，我们总结了RNA靶标筛选方法的最新进展，并概述了针对各种类别和治疗领域的RNA靶标的新型药物样小分子的发现。结构，功能和小分子可药性之间的联系现在首次证明RNA可以成为治疗药物的靶标。