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Nucleolar GTP-binding protein 1 (GTPBP4) is a conserved GTPase located predominantly in the nucleolus, encoded by the GTPBP4 gene on human chromosome 10. It functions as a molecular switch cycling between GTP- and GDP-bound states, playing an essential regulatory role in the biogenesis of the 60S ribosomal subunit, which is crucial for protein synthesis and cellular proliferation[1][2][3][4][6]. GTPBP4 also influences cell cycle progression via repression of the TP53 pathway and has been implicated in cancer biology: it is upregulated in several cancer types, including non-small cell lung cancer and liver cancer, where its high expression promotes cell proliferation, invasion, and tumor growth—likely via regulation of processes such as EMT (epithelial-mesenchymal transition)[2][3]. While not currently a drug target with known small-molecule modulators, GTPBP4 is under investigation as a potential therapeutic target and biomarker in oncology[2]. Notes: - No approved drugs or inhibitors currently in clinical use or trial specifically targeting GTPBP4 could be identified in the provided results. - Most disease roles currently focus on cancer biology, with evidence from both cell line and animal studies[2].
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