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Targeting RNA structures in diseases with small molecules.

Authors
  • Shao, Yanqiu1, 2
  • Zhang, Qiangfeng Cliff1, 2
  • 1 MOE Key Laboratory of Bioinformatics, Beijing Advanced Innovation Center for Structural Biology and Frontier Research Center for Biological Structure, Center for Synthetic and Systems Biology, School of Life Sciences, Tsinghua University, Beijing 100084, China. , (China)
  • 2 Tsinghua-Peking Center for Life Sciences, Beijing 100084, China. , (China)
Type
Published Article
Journal
Essays in biochemistry
Publication Date
Dec 07, 2020
Volume
64
Issue
6
Pages
955–966
Identifiers
DOI: 10.1042/EBC20200011
PMID: 33078198
Source
Medline
Keywords
Language
English
License
Unknown

Abstract

RNA is crucial for gene expression and regulation. Recent advances in understanding of RNA biochemistry, structure and molecular biology have revealed the importance of RNA structure in cellular processes and diseases. Various approaches to discovering drug-like small molecules that target RNA structure have been developed. This review provides a brief introduction to RNA structural biology and how RNA structures function as disease regulators. We summarize approaches to targeting RNA with small molecules and highlight their advantages, shortcomings and therapeutic potential. © 2020 The Author(s).

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