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Functionally active modulators targeting the LRRK2 WD40 repeat domain identified by FRASE-bot in CACHE Challenge #1

Title: Functionally active modulators targeting the LRRK2 WD40 repeat domain identified by FRASE-bot in CACHE Challenge #1
Authors: Mettu, Akhila; Glavatskikh, Marta; Wang, Xiaowen; Lara Ordonez, Antonio Jesus; Li, Fengling; Chau, Irene; Ackloo, Suzanne; Arrowsmith, Cheryl; Bolotokova, Albina; Ghiabi, Pegah; Gibson, Elisa; Halabelian, Levon; Houliston, Scott; Harding, Rachel J.; Hutchinson, Ashley; Loppnau, Peter; Perveen, Sumera; Seitova, Almagul; Zeng, Hong; Schapira, Matthieu; Taymans, Jean-Marc; Kireev, Dmitri
Contributors: Université de Lille; Inserm; CHU Lille; University of Missouri Columbia Mizzou; University of North Carolina at Chapel Hill NC, USA UNC; Lille Neurosciences & Cognition - U 1172 LilNCog; Structural Genomics Consortium; University of Toronto; Princess Margaret Hospital; Department of Pharmacology and Toxicology Toronto; Lille Neurosciences & Cognition (LilNCog) - U 1172
Publisher Information: The Royal Society of Chemistry
Publication Year: 2025
Collection: LillOA (Lille Open Archive - Université de Lille)
Description: Critical Assessment of Computational Hit-Finding Experiments (CACHE) Challenges emerged as real-life stress tests for computational hit-finding strategies. In CACHE Challenge #1, 23 participants contributed their original workflows to identify small-molecule ligands for the WD40 repeat (WDR) of LRRK2, a promising Parkinson's target. We applied the FRASE-based hit-finding robot (FRASE-bot), a platform for interaction-based screening allowing a drastic reduction of the explorable chemical space and a concurrent detection of putative ligand-binding sites. In two screening rounds, 84 compounds were procured for experimental testing and 8 were confirmed to bind LRRK2-WDR with dissociation constants (Kd) ranging from 3 to 41 μM. To investigate the functional effect of WDR ligands, they were tested for their ability to modify the LRRK2 activity markers in HEK293T cells. Two compounds showed statistically significant increases in the kinase activity of WT LRRK2, and two compounds affected the conformation and kinase activity of major LRRK2 mutants. ; 16;8
Document Type: article in journal/newspaper
File Description: application/octet-stream; application/rdf+xml; charset=utf-8; application/pdf
Language: English
Relation: PARK-PEP: Ciblage de la phosphorylation de LRRK2 par peptides interférents en modèles experimentaux de la maladie de Parkinson; Chemical Science; Chem. Sci.; http://hdl.handle.net/20.500.12210/126579
Availability: https://hdl.handle.net/20.500.12210/126579
Rights: Attribution-NonCommercial 3.0 United States ; info:eu-repo/semantics/openAccess
Accession Number: edsbas.CBA9CA53
Database: BASE