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 …

RNA drugs and RNA targets for small molecules: principles, progress, and challenges

AM Yu, YH Choi, MJ Tu - Pharmacological reviews, 2020 - ASPET
RNA-based therapies, including RNA molecules as drugs and RNA-targeted small
molecules, offer unique opportunities to expand the range of therapeutic targets. Various …

[HTML][HTML] Principles for targeting RNA with drug-like small molecules

KD Warner, CE Hajdin, KM Weeks - Nature reviews Drug discovery, 2018 - nature.com
RNA molecules are essential for cellular information transfer and gene regulation, and
RNAs have been implicated in many human diseases. Messenger and non-coding RNAs …

The roles of structural dynamics in the cellular functions of RNAs

LR Ganser, ML Kelly, D Herschlag… - … reviews Molecular cell …, 2019 - nature.com
RNAs fold into 3D structures that range from simple helical elements to complex tertiary
structures and quaternary ribonucleoprotein assemblies. The functions of many regulatory …

Targeting RNA with small molecules: from fundamental principles towards the clinic

JP Falese, A Donlic, AE Hargrove - Chemical Society Reviews, 2021 - pubs.rsc.org
Recent advances in our understanding of RNA biology have uncovered crucial roles for
RNA in multiple disease states, ranging from viral and bacterial infections to cancer and …

Targeting Xist with compounds that disrupt RNA structure and X inactivation

R Aguilar, KB Spencer, B Kesner, NF Rizvi… - Nature, 2022 - nature.com
Although more than 98% of the human genome is non-coding, nearly all of the drugs on the
market target one of about 700 disease-related proteins. The historical reluctance to invest …

Small-molecule modulation of TDP-43 recruitment to stress granules prevents persistent TDP-43 accumulation in ALS/FTD

MY Fang, S Markmiller, AQ Vu, A Javaherian… - Neuron, 2019 - cell.com
Stress granules (SGs) form during cellular stress and are implicated in neurodegenerative
diseases such as amyotrophic lateral sclerosis and frontotemporal dementia (ALS/FTD). To …

Targeting RNA with small molecules to capture opportunities at the intersection of chemistry, biology, and medicine

MD Disney - Journal of the American Chemical Society, 2019 - ACS Publications
The biology of healthy and disease-affected cells is often mediated by RNA structures,
desirable targets for small molecule chemical probes and lead medicines. Although …

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 …

The emerging role of RNA as a therapeutic target for small molecules

CM Connelly, MH Moon, JS Schneekloth - Cell chemical biology, 2016 - cell.com
Recent advances in understanding different RNAs and unique features of their biology have
revealed a wealth of information. However, approaches to identify small molecules that …