Division of Nephrology, Department of Medicine, New York Medical College, Valhalla, NY, USA.
Endocrine Unit and Pediatric Nephrology Unit, Massachusetts General Hospital, Boston, MA, USA; Harvard Medical School, Boston, MA, USA.
Best Pract Res Clin Endocrinol Metab. 2018 Oct;32(5):685-706. doi: 10.1016/j.beem.2018.06.004. Epub 2018 Jun 18.
Our understanding of the regulation of phosphate balance has benefited tremendously from the molecular identification and characterization of genetic defects leading to a number of rare inherited or acquired disorders affecting phosphate homeostasis. The identification of the key phosphate-regulating hormone, fibroblast growth factor 23 (FGF23), as well as other molecules that control its production, such as the glycosyltransferase GALNT3, the endopeptidase PHEX, and the matrix protein DMP1, and molecules that function as downstream effectors of FGF23 such as the longevity factor Klotho and the phosphate transporters NPT2a and NPT2c, has permitted us to understand the complex interplay that exists between the kidneys, bone, parathyroid, and gut. Such insights from genetic disorders have allowed not only the design of potent targeted treatment of FGF23-dependent hypophosphatemic conditions, but also provide clinically relevant observations related to the dysregulation of mineral ion homeostasis in health and disease.
我们对磷酸盐平衡调控的理解得益于对导致一系列影响磷酸盐稳态的罕见遗传性或获得性疾病的遗传缺陷的分子鉴定和特征描述。关键磷酸盐调节激素成纤维细胞生长因子 23(FGF23)的鉴定,以及控制其产生的其他分子,如糖基转移酶 GALNT3、内肽酶 PHEX 和基质蛋白 DMP1,以及作为 FGF23 的下游效应物的分子,如长寿因子 Klotho 和磷酸盐转运体 NPT2a 和 NPT2c,使我们能够理解肾脏、骨骼、甲状旁腺和肠道之间存在的复杂相互作用。这些遗传疾病的见解不仅允许设计针对 FGF23 依赖性低磷酸盐血症的有效靶向治疗,还提供了与健康和疾病中矿物质离子稳态失调相关的临床相关观察结果。