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Targeting RNA with small molecules: from fundamental principles towards the clinic by James P. Falese; Anita Donlic; Amanda E. Hargrove is a Chemistry article available to read on EtoBox.

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Recent advances in our understanding of RNA biology have uncovered crucial roles for RNA in multiple disease states, ranging from viral and bacterial infections to cancer and neurological disorders. As a result, multiple laboratories have become interested in developing drug-like small molecules to target RNA. However, this development comes with multiple unique challenges. For example, RNA is inherently dynamic and has limited chemical diversity. In addition, promiscuous RNA-binding ligands are often identified during screening campaigns. This Tutorial Review overviews important considerations and advancements for generating RNA-targeted small molecules, ranging from fundamental chemistry to promising small molecule examples with demonstrated clinical efficacy. Specifically, we begin by exploring RNA functional classes, structural hierarchy, and dynamics. We then discuss fundamental RNA recognition principles along with methods for small molecule screening and RNA structure determination. Finally, we review unique challenges and emerging solutions from both the RNA and small molecule perspectives for generating RNA-targeted ligands before highlighting a selection of the ''Greatest

Who reads Targeting RNA with small molecules: from fundamental principles towards the clinic?

Author

James P. Falese; Anita Donlic; Amanda E. Hargrove

Publisher

Royal Society of Chemistry (RSC)

Published

2021

Language

EN

Field

Chemistry (Physical Sciences)