Beijing Frontier Research Center for Biological Structure, Tsinghua-Peking Joint Center for Life Sciences, State Key Laboratory of Membrane Biology, School of Life Sciences, Tsinghua University, 100084, Beijing, China.
Institute of Bio-Architecture and Bio-Interactions (IBABI), Shenzhen Medical Academy of Research and Translation (SMART), Shenzhen, 518107, Guangdong, China.
Nat Commun. 2024 Jun 12;15(1):5039. doi: 10.1038/s41467-024-49420-9.
Urate, the physiological form of uric acid and a potent antioxidant in serum, plays a pivotal role in scavenging reactive oxygen species. Yet excessive accumulation of urate, known as hyperuricemia, is the primary risk factor for the development of gout. The high-capacity urate transporter GLUT9 represents a promising target for gout treatment. Here, we present cryo-electron microscopy structures of human GLUT9 in complex with urate or its inhibitor apigenin at overall resolutions of 3.5 Å and 3.3 Å, respectively. In both structures, GLUT9 exhibits an inward open conformation, wherein the substrate binding pocket faces the intracellular side. These structures unveil the molecular basis for GLUT9's substrate preference of urate over glucose, and show that apigenin acts as a competitive inhibitor by occupying the substrate binding site. Our findings provide critical information for the development of specific inhibitors targeting GLUT9 as potential therapeutics for gout and hyperuricemia.
尿酸是尿酸的生理形式,也是血清中的一种强效抗氧化剂,在清除活性氧方面发挥着关键作用。然而,尿酸的过度积累,即高尿酸血症,是痛风发展的主要危险因素。高尿酸转运蛋白 GLUT9 是治疗痛风的一个很有前途的靶点。在这里,我们分别以 3.5Å 和 3.3Å 的整体分辨率呈现了与人 GLUT9 复合物的尿酸或其抑制剂芹菜素的冷冻电子显微镜结构。在这两种结构中,GLUT9 均呈现出内向开放的构象,其中底物结合口袋朝向细胞内一侧。这些结构揭示了 GLUT9 对尿酸而非葡萄糖的底物偏好的分子基础,并表明芹菜素通过占据底物结合位点充当竞争性抑制剂。我们的研究结果为开发针对 GLUT9 的特异性抑制剂提供了关键信息,这些抑制剂可能成为治疗痛风和高尿酸血症的潜在疗法。