Heterobifunctional small molecules to modulate RNA function

S Kovachka, Y Tong, JL Childs-Disney… - Trends in …, 2024 - cell.com
RNA has diverse cellular functionality, including regulating gene expression, protein
translation, and cellular response to stimuli, due to its intricate structures. Over the past …

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 …

Targeting RNA with small molecules—A safety perspective

HL Lightfoot, GF Smith - British Journal of Pharmacology, 2023 - Wiley Online Library
RNA is a major player in cellular function, and consequently can drive a number of disease
pathologies. Over the past several years, small molecule‐RNA targeting (smRNA targeting) …

Drug-like small molecules that inhibit expression of the oncogenic microRNA-21

MD Shortridge, B Chaubey, HJ Zhang… - ACS chemical …, 2023 - ACS Publications
We report the discovery of drug-like small molecules that bind specifically to the precursor of
the oncogenic and pro-inflammatory microRNA-21 with mid-nanomolar affinity. The small …

LncRNA IL21-AS1 facilitates tumour progression by enhancing CD24-induced phagocytosis inhibition and tumorigenesis in ovarian cancer

J Liu, C Yan, S Xu - Cell Death & Disease, 2024 - nature.com
CD24 is overexpressed in various tumours and considered a regulator of cell migration,
invasion, and proliferation. Recent studies have found that CD24 on ovarian cancer (OC) …

Key considerations in targeted protein degradation drug discovery and development

L Qin, H Dai, J Wang - Frontiers in Chemistry, 2022 - frontiersin.org
Targeting proteins' enzymatic functions with small molecule inhibitors, as well as functions of
receptor proteins with small-molecule agonists and antagonists, were the major forms of …

De‐Novo Design of pre‐miR‐21 Maturation Inhibitors: Synthesis and Activity Assessment

I Shcheholeva, D Fernández‐Remacha… - … A European Journal, 2023 - Wiley Online Library
Targeting RNA with small molecules is a major challenge of current medicinal chemistry,
and the identification and design of original scaffolds able to selectively interact with an RNA …

[HTML][HTML] Small-molecule screening of ribonuclease L binders for RNA degradation

L Borgelt, N Haacke, P Lampe, X Qiu, R Gasper… - Biomedicine & …, 2022 - Elsevier
Small molecules targeting the ubiquitous latent ribonuclease (RNase L), which has limited
sequence specificity toward single-stranded RNA substrates, hold great potential to be …

Bifunctional chimera for ligand-directed photo-degradation of oncogenic microRNA

C Li, J Wang, Y Wang, Y Feng, J Li… - Chemical …, 2023 - pubs.rsc.org
Targeted inhibition of oncogenic microRNAs provides a promising anti-cancer approach.
Here, we report a bifunctional chimera for ligand-directed regulation of target oncogenic …

Responsive MXene nanovehicles deliver CRISPR/Cas12a for boolean logic-controlled gene editing

S Wang, Z Zhang, R Tang, F He, Y Huang, Z Nie… - Science China …, 2022 - Springer
Programmable and precise regulation of genetic information is crucial in bioengineering and
biomedicine; however, it remains challenging to implement this objective. Here we deployed …