Drugging the RNA World

SUMMARYAlthough we live in the remnants of an RNA world, the world of drug discovery and chemical probes is firmly protein-centric. Developing highly selective small molecules targeting RNA is often considered to be an insurmountable challenge. Our goal is to demystify the design of such compounds....

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Published inCold Spring Harbor perspectives in biology Vol. 10; no. 11; p. a034769
Main Authors Disney, Matthew D, Dwyer, Brendan G, Childs-Disney, Jessica L
Format Journal Article
LanguageEnglish
Published United States Cold Spring Harbor Laboratory Press 01.11.2018
Subjects
Biology
CONCEPT
Design
DNA probes
Drug discovery
Nucleotide sequence
Organic chemistry
Probes
Proteins
Ribonucleic acid
RNA
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Summary:SUMMARYAlthough we live in the remnants of an RNA world, the world of drug discovery and chemical probes is firmly protein-centric. Developing highly selective small molecules targeting RNA is often considered to be an insurmountable challenge. Our goal is to demystify the design of such compounds. In this review, we describe various approaches to design small molecules that target RNA from sequence and the application of these compounds in RNA biology, with a focus on inhibition of human RNA-protein complexes. We have developed a library-versus-library screening approach to define selective RNA-small-molecule binding partners and applied them to disease-causing RNAs, in particular noncoding oncogenic RNAs and expanded RNA repeats, to modulate their biology in cells and animals. We also describe the design of new types of small-molecule probes that could broadly decipher the mysteries of RNA in cells.
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ISSN:1943-0264
1943-0264
DOI:10.1101/cshperspect.a034769