[HTML][HTML] Emerging concepts of miRNA therapeutics: from cells to clinic

C Diener, A Keller, E Meese - Trends in Genetics, 2022 - cell.com
MicroRNAs (miRNAs) are very powerful genetic regulators, as evidenced by the fact that a
single miRNA can direct entire cellular pathways via interacting with a broad spectrum of …

Noncoding RNA therapeutics—challenges and potential solutions

M Winkle, SM El-Daly, M Fabbri, GA Calin - Nature reviews Drug …, 2021 - nature.com
Therapeutic targeting of noncoding RNAs (ncRNAs), such as microRNAs (miRNAs) and
long noncoding RNAs (lncRNAs), represents an attractive approach for the treatment of …

[HTML][HTML] Programming inactive RNA-binding small molecules into bioactive degraders

Y Tong, Y Lee, X Liu, JL Childs-Disney, BM Suresh… - Nature, 2023 - nature.com
Target occupancy is often insufficient to elicit biological activity, particularly for RNA,
compounded by the longstanding challenges surrounding the molecular recognition of RNA …

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 …

Amplifying gene expression with RNA-targeted therapeutics

O Khorkova, J Stahl, A Joji, CH Volmar… - Nature Reviews Drug …, 2023 - nature.com
Many diseases are caused by insufficient expression of mutated genes and would benefit
from increased expression of the corresponding protein. However, in drug development, it …

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 …

Non-coding RNAs in cancer: Platforms and strategies for investigating the genomic “dark matter”

K Grillone, C Riillo, F Scionti, R Rocca… - Journal of Experimental …, 2020 - Springer
The discovery of the role of non-coding RNAs (ncRNAs) in the onset and progression of
malignancies is a promising frontier of cancer genetics. It is clear that ncRNAs are …

Small molecule recognition of disease-relevant RNA structures

SM Meyer, CC Williams, Y Akahori, T Tanaka… - Chemical Society …, 2020 - pubs.rsc.org
Targeting RNAs with small molecules represents a new frontier in drug discovery and
development. The rich structural diversity of folded RNAs offers a nearly unlimited reservoir …

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 …

How we think about targeting RNA with small molecules

MG Costales, JL Childs-Disney, HS Haniff… - Journal of medicinal …, 2020 - ACS Publications
RNA offers nearly unlimited potential as a target for small molecule chemical probes and
lead medicines. Many RNAs fold into structures that can be selectively targeted with small …