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ML141 is a potent, selective, and reversible small molecule inhibitor of the Rho family GTPase Cdc42. It acts as a non-competitive inhibitor that prevents the binding of GTP to Cdc42, thereby blocking its activation and downstream signaling pathways involved in actin cytoskeleton remodeling, cell migration, and vesicle trafficking. Originally identified through the NIH Molecular Libraries Probe Production Centers Network (MLPCN), ML141 is widely used as a chemical probe in cell biology research. Recent preclinical studies have explored its therapeutic potential in inflammatory conditions such as ANCA-associated vasculitis (AAV), where it has been shown to inhibit pathogenic neutrophil effector mechanisms, including NETosis and actin polymerization, and reduce glomerular leukocyte infiltration in animal models.
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