Targeting RNA structures with small molecules

JL Childs-Disney, X Yang, QMR Gibaut… - Nature Reviews Drug …, 2022 - nature.com
RNA adopts 3D structures that confer varied functional roles in human biology and
dysfunction in disease. Approaches to therapeutically target RNA structures with small …

Mass spectrometry of nucleic acid noncovalent complexes

E Largy, A Konig, A Ghosh, D Ghosh… - Chemical …, 2021 - ACS Publications
Nucleic acids have been among the first targets for antitumor drugs and antibiotics. With the
unveiling of new biological roles in regulation of gene expression, specific DNA and RNA …

DNA-binding of nickel (II), copper (II) and zinc (II) complexes: Structure–affinity relationships

G Barone, A Terenzi, A Lauria, AM Almerico… - Coordination Chemistry …, 2013 - Elsevier
Abstract Nickel (II), copper (II) and zinc (II) complexes with the same ligands normally display
analogous coordination geometry and binding mode toward DNA. However, although …

Contemporary progress and opportunities in RNA-targeted drug discovery

AL Garner - ACS Medicinal Chemistry Letters, 2023 - ACS Publications
The surprising discovery that RNAs are the predominant gene products to emerge from the
human genome catalyzed a renaissance in RNA biology. It is now well-understood that …

Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes

A Garcia-Lopez, F Tessaro, HRA Jonker… - Nature …, 2018 - nature.com
Modification of SMN2 exon 7 (E7) splicing is a validated therapeutic strategy against spinal
muscular atrophy (SMA). However, a target-based approach to identify small-molecule E7 …

Small molecule targeting r(UGGAA)n disrupts RNA foci and alleviates disease phenotype in Drosophila model

T Shibata, K Nagano, M Ueyama, K Ninomiya… - Nature …, 2021 - nature.com
Synthetic small molecules modulating RNA structure and function have therapeutic potential
for RNA diseases. Here we report our discovery that naphthyridine carbamate dimer (NCD) …

19F NMR‐Based Fragment Screening for 14 Different Biologically Active RNAs and 10 DNA and Protein Counter‐Screens

O Binas, V de Jesus, T Landgraf, AE Völklein… - …, 2021 - Wiley Online Library
We report here the nuclear magnetic resonance 19F screening of 14 RNA targets with
different secondary and tertiary structure to systematically assess the druggability of RNAs …

Fluorescent indicator displacement assays to identify and characterize small molecule interactions with RNA

SL Wicks, AE Hargrove - Methods, 2019 - Elsevier
Fluorescent indicator displacement (FID) assays are an advantageous approach to convert
receptors into optical sensors that can detect binding of various ligands. In particular, the …

Unveiling the druggable RNA targets and small molecule therapeutics

J Sztuba-Solinska, G Chavez-Calvillo… - Bioorganic & medicinal …, 2019 - Elsevier
The increasing appreciation for the crucial roles of RNAs in infectious and non-infectious
human diseases makes them attractive therapeutic targets. Coding and non-coding RNAs …

Identifying the preferred RNA motifs and chemotypes that interact by probing millions of combinations

T Tran, MD Disney - Nature communications, 2012 - nature.com
RNA is an important therapeutic target but information about RNA–ligand interactions is
limited. Here, we report a screening method that probes over 3,000,000 combinations of …