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

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

Author information

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.

PMID:
31356880
PMCID:
PMC6842402
[Available on 2020-10-01]
DOI:
10.1016/j.drudis.2019.06.019

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