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Synthetic RNA Modulators in Drug Discovery

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Review Chemistry, Multidisciplinary

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

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Summary: This article reviews recent advances in understanding RNA biology, including its crucial roles in various disease states and the challenges and methods for drug design targeting RNA. It focuses on discussing RNA structural hierarchy, functional classes, and dynamics, as well as methods for small molecule screening and RNA structure determination. Finally, it summarizes the unique challenges and emerging solutions for generating RNA-targeted ligands from both RNA and small molecule perspectives.

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