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 …

Tapping the RNA world for therapeutics

J Lieberman - Nature structural & molecular biology, 2018 - nature.com
A recent revolution in RNA biology has led to the identification of new RNA classes with
unanticipated functions, new types of RNA modifications, an unexpected multiplicity of …

Synthetic RNA modulators in drug discovery

F Zamani, T Suzuki - Journal of Medicinal Chemistry, 2021 - ACS Publications
RNAs are involved in an enormous range of cellular processes, including gene regulation,
protein synthesis, and cell differentiation, and dysfunctional RNAs are associated with …

RNA as a small molecule druggable target

NF Rizvi, GF Smith - Bioorganic & Medicinal Chemistry Letters, 2017 - Elsevier
Small molecule drugs have readily been developed against many proteins in the human
proteome, but RNA has remained an elusive target for drug discovery. Increasingly, we see …

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 …

[HTML][HTML] Screening strategies for identifying RNA-and ribonucleoprotein-targeted compounds

WJ Martin, P Grandi, M Marcia - Trends in Pharmacological Sciences, 2021 - cell.com
The past few years have witnessed important breakthroughs in the identification of
compounds that specifically bind and regulate RNAs and in optimizing them for therapeutic …

[HTML][HTML] 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 …

RNA–Small-Molecule Interaction: Challenging the “Undruggable” Tag

J Kaur, A Sharma, P Mundlia, V Sood… - Journal of Medicinal …, 2024 - ACS Publications
RNA targeting, specifically with small molecules, is a relatively new and rapidly emerging
avenue with the promise to expand the target space in the drug discovery field. From being …

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 …

Small molecule–RNA targeting: starting with the fundamentals

AE Hargrove - Chemical Communications, 2020 - pubs.rsc.org
The structural and regulatory elements in therapeutically relevant RNAs offer many
opportunities for targeting by small molecules, yet fundamental understanding of what drives …