Methods to identify and optimize small molecules interacting with RNA (SMIRNAs)

Andrei Ursu; Simon Vézina-Dawod; Matthew D Disney

doi:10.1016/j.drudis.2019.06.019

Methods to identify and optimize small molecules interacting with RNA (SMIRNAs)

Drug Discov Today. 2019 Oct;24(10):2002-2016. doi: 10.1016/j.drudis.2019.06.019. Epub 2019 Jul 26.

Authors

Andrei Ursu¹, Simon Vézina-Dawod¹, Matthew D Disney²

Affiliations

¹ Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.
² Department of Chemistry, The Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA. Electronic address: disney@scripps.edu.

Abstract

RNAs, particularly noncoding RNAs (ncRNAs), are becoming increasingly important therapeutic targets, because they are causative and antagonists of human disease. Indeed, aberrant RNA structural elements and expression deregulate biological processes. In this review, we describe methodologies to discover and optimize small molecules interacting with RNA (SMIRNAs), including the evaluation of direct target engagement and the rescue of RNA-mediated phenotypes in vitro and in vivo. Such studies are essential to fully characterize the mode of action of SMIRNAs and advance our understanding of rationally and efficiently drugging RNAs for therapeutic benefit.

Publication types

Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Review

MeSH terms

Drug Discovery / methods*
Humans
Microarray Analysis / methods
RNA, Untranslated / adverse effects
RNA, Untranslated / drug effects*
Small Molecule Libraries / therapeutic use*

Substances

RNA, Untranslated
Small Molecule Libraries

Abstract

Publication types

MeSH terms

Substances

Grants and funding