期刊
BIOORGANIC & MEDICINAL CHEMISTRY LETTERS
卷 27, 期 23, 页码 5083-5088出版社
PERGAMON-ELSEVIER SCIENCE LTD
DOI: 10.1016/j.bmcl.2017.10.052
关键词
Non-coding RNA; ncRNA; Drug target; Small molecule; Target validation; Functional
资金
- Merck & Co. Inc., Kenilworth, New Jersey
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. (C) 2017 Elsevier Ltd. All rights reserved.
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