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WD repeat domain 24 (WDR24) is a **protein-coding gene** that encodes a catalytic component of the GATOR2 complex, which is a multi-protein assembly involved in activation of the amino acid-sensing branch of the **mTORC1 signaling pathway**. WDR24 participates primarily in regulating cell growth, metabolism, and autophagy through interaction with the GATOR2 and GATOR1 complexes, serving as a scaffold and E3 ubiquitin ligase subunit within GATOR2[1][2]. The protein contains WD-repeat domains that typically form a beta-propeller structure, facilitating protein-protein interactions but not acting as a classical enzyme or receptor[1][3][6]. Mutations in WDR24 have been associated with rare genetic disorders such as **mosaic variegated aneuploidy syndrome 1** and may have broader implications in cellular stress and metabolic signaling, but it is not a recognized therapeutic drug target or clinical biomarker at this time[1]. **Notes:** - WDR24 is not considered a classical therapeutic target (e.g., not a receptor, enzyme, or transport protein), but plays a regulatory role in key signaling pathways relevant to disease biology. - No drugs are known or reported to directly target WDR24; most drug efforts in the mTOR pathway target mTOR or related kinases rather than scaffold components like GATOR2. - WD-repeat proteins serve structural and complex-assembly functions rather than catalytic roles; WDR24's E3 ubiquitin ligase activity operates in the context of the multiprotein complex, rather than as an isolated enzyme[1][2][6].
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