Anyatonwu Georgia I, Ehrlich Barbara E
Department of Pharmacology, Yale University School of Medicine, New Haven, CT, USA.
Biochem Biophys Res Commun. 2004 Oct 1;322(4):1364-73. doi: 10.1016/j.bbrc.2004.08.043.
Polycystic kidney disease (PKD) is caused by mutations in two genes, PKD1 and PKD2, which encode for the proteins, polycystin-1 (PC1) and polycystin-2 (PC2), respectively. Although disease-associated mutations have been identified in these two proteins, the sequence of molecular events leading up to clinical symptoms is still unknown. PC1 resides in the plasma membrane and it is thought to function in cell-cell and cell-matrix interactions, whereas PC2 is a calcium (Ca2+) permeable cation channel concentrated in the endoplasmic reticulum. Both proteins localize to the primary cilia where they function as a mechanosensitive receptor complex allowing the entry of Ca2+ into the cell. The downstream signaling pathway involves activation of intracellular Ca2+ release channels, especially the ryanodine receptor (RyR), but subsequent steps are still to be identified. Elucidation of the signaling pathway involved in normal PC1/PC2 function, the functional consequences of PC1/PC2 mutation, and the role of Ca2+ signaling will all help to unravel the molecular mechanisms of cystogenesis in PKD.
多囊肾病(PKD)由两个基因PKD1和PKD2的突变引起,这两个基因分别编码蛋白质多囊蛋白-1(PC1)和多囊蛋白-2(PC2)。尽管已在这两种蛋白质中鉴定出与疾病相关的突变,但导致临床症状的分子事件序列仍不清楚。PC1存在于质膜中,被认为在细胞间和细胞与基质的相互作用中发挥作用,而PC2是一种钙(Ca2+)可渗透的阳离子通道,集中在内质网中。这两种蛋白质都定位于初级纤毛,在那里它们作为机械敏感受体复合物发挥作用,允许Ca2+进入细胞。下游信号通路涉及细胞内Ca2+释放通道的激活,尤其是兰尼碱受体(RyR),但后续步骤仍有待确定。阐明正常PC1/PC2功能所涉及的信号通路、PC1/PC2突变的功能后果以及Ca2+信号的作用,都将有助于揭示PKD中囊肿形成的分子机制。