Small molecule approaches to targeting RNA
S Kovachka, M Panosetti, B Grimaldi… - Nature Reviews …, 2024 - nature.com
The development of innovative methodologies to identify RNA binders has attracted
enormous attention in chemical biology and drug discovery. Although antibiotics targeting …
enormous attention in chemical biology and drug discovery. Although antibiotics targeting …
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 …
human genome catalyzed a renaissance in RNA biology. It is now well-understood that …
DNA-encoded library screening to inform design of a ribonuclease targeting chimera (RiboTAC)
SM Meyer, T Tanaka, PRA Zanon… - Journal of the …, 2022 - ACS Publications
Ribonuclease targeting chimeras (RiboTACs) induce degradation of an RNA target by
facilitating an interaction between an RNA and a ribonuclease (RNase). We describe the …
facilitating an interaction between an RNA and a ribonuclease (RNase). We describe the …
Pervasive transcriptome interactions of protein-targeted drugs
The off-target toxicity of drugs targeted to proteins imparts substantial health and economic
costs. Proteome interaction studies can reveal off-target effects with unintended proteins; …
costs. Proteome interaction studies can reveal off-target effects with unintended proteins; …
Transcriptome-Wide Studies of RNA-Targeted Small Molecules Provide a Simple and Selective r(CUG)exp Degrader in Myotonic Dystrophy
QMR Gibaut, JA Bush, Y Tong, JT Baisden… - ACS Central …, 2023 - ACS Publications
Myotonic dystrophy type 1 (DM1) is caused by a highly structured RNA repeat expansion, r
(CUG) exp, harbored in the 3′ untranslated region (3′ UTR) of dystrophia myotonica …
(CUG) exp, harbored in the 3′ untranslated region (3′ UTR) of dystrophia myotonica …
Study of an RNA-focused DNA-encoded library informs design of a degrader of ar (CUG) repeat expansion
QMR Gibaut, Y Akahori, JA Bush… - Journal of the …, 2022 - ACS Publications
A solid-phase DNA-encoded library (DEL) was studied for binding the RNA repeat
expansion r (CUG) exp, the causative agent of the most common form of adult-onset …
expansion r (CUG) exp, the causative agent of the most common form of adult-onset …
Targeting ribonucleases with small molecules and bifunctional molecules
L Borgelt, P Wu - ACS Chemical Biology, 2023 - ACS Publications
Ribonucleases (RNases) cleave and process RNAs, thereby regulating the biogenesis,
metabolism, and degradation of coding and noncoding RNAs. Thus, small molecules …
metabolism, and degradation of coding and noncoding RNAs. Thus, small molecules …
Fragment-based approaches to identify RNA binders
Although fragment-based drug discovery (FBDD) has been successfully implemented and
well-explored for protein targets, its feasibility for RNA targets is emerging. Despite the …
well-explored for protein targets, its feasibility for RNA targets is emerging. Despite the …
Discovery of RNA-targeted small molecules through the merging of experimental and computational technologies
EC Morishita - Expert Opinion on Drug Discovery, 2023 - Taylor & Francis
Introduction The field of RNA-targeted small molecules is rapidly evolving, owing to the
advances in experimental and computational technologies. With the identification of several …
advances in experimental and computational technologies. With the identification of several …
Low-molecular weight small molecules can potently bind RNA and affect oncogenic pathways in cells
RNA is challenging to target with bioactive small molecules, particularly those of low
molecular weight that bind with sufficient affinity and specificity. In this report, we developed …
molecular weight that bind with sufficient affinity and specificity. In this report, we developed …