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Integrating fragment-based screening with targeted protein degradation and genetic rescue to explore eIF4E function.

Title: Integrating fragment-based screening with targeted protein degradation and genetic rescue to explore eIF4E function.
Authors: Sharp, SY; Martella, M; D'Agostino, S; Milton, CI; Ward, G; Woodhead, AJ; Richardson, CJ; Carr, MG; Chiarparin, E; Cons, BD; Coyle, J; East, CE; Hiscock, SD; Martinez-Fleites, C; Mortenson, PN; Palmer, N; Pathuri, P; Powers, MV; Saalau, SM; St Denis, JD; Swabey, K; Vinković, M; Walton, H; Williams, G; Clarke, PA
Contributors: Powers, Marissa; Clarke, Paul
Publisher Information: NATURE PORTFOLIO
Publication Year: 2025
Collection: The Institute of Cancer Research (ICR): Publications Repository
Subject Terms: Eukaryotic Initiation Factor-4E; Humans; Eukaryotic Initiation Factor-4G; Binding Sites; Proteolysis; Protein Binding; Protein Biosynthesis; HEK293 Cells; Drug Discovery; Small Molecule Libraries; Ligands
Subject Geographic: England
Description: Eukaryotic initiation factor 4E (eIF4E) serves as a regulatory hub for oncogene-driven protein synthesis and is considered a promising anticancer target. Here we screen a fragment library against eIF4E and identify a ligand-binding site with previously unknown function. Follow-up structure-based design yields a low nM tool compound (4, Kd = 0.09 µM; LE 0.38), which disrupts the eIF4E:eIF4G interaction, inhibits translation in cell lysates, and demonstrates target engagement with eIF4E in intact cells (EC50 = 2 µM). By coupling targeted protein degradation with genetic rescue using eIF4E mutants, we show that disruption of both the canonical eIF4G and non-canonical binding sites is likely required to drive a strong cellular effect. This work highlights the power of fragment-based drug discovery to identify pockets in difficult-to-drug proteins and how this approach can be combined with genetic characterization and degrader technology to probe protein function in complex biological systems.
Document Type: article in journal/newspaper
File Description: Electronic; application/pdf
Language: English
ISSN: 2041-1723
Relation: ARTN 10037; Nature Communications, 2024, 15 (1), pp. 10037 -; https://repository.icr.ac.uk/handle/internal/6790
Availability: https://repository.icr.ac.uk/handle/internal/6790
Rights: http://creativecommons.org/licenses/by/4.0/
Accession Number: edsbas.B9FED592
Database: BASE