Nauli Surya M, Alenghat Francis J, Luo Ying, Williams Eric, Vassilev Peter, Li Xiaogang, Elia Andrew E H, Lu Weining, Brown Edward M, Quinn Stephen J, Ingber Donald E, Zhou Jing
Renal Division, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, 4 Blackfan Circle, Boston, Massachusetts 02115, USA.
Nat Genet. 2003 Feb;33(2):129-37. doi: 10.1038/ng1076. Epub 2003 Jan 6.
Several proteins implicated in the pathogenesis of polycystic kidney disease (PKD) localize to cilia. Furthermore, cilia are malformed in mice with PKD with mutations in TgN737Rpw (encoding polaris). It is not known, however, whether ciliary dysfunction occurs or is relevant to cyst formation in PKD. Here, we show that polycystin-1 (PC1) and polycystin-2 (PC2), proteins respectively encoded by Pkd1 and Pkd2, mouse orthologs of genes mutated in human autosomal dominant PKD, co-distribute in the primary cilia of kidney epithelium. Cells isolated from transgenic mice that lack functional PC1 formed cilia but did not increase Ca(2+) influx in response to physiological fluid flow. Blocking antibodies directed against PC2 similarly abolished the flow response in wild-type cells as did inhibitors of the ryanodine receptor, whereas inhibitors of G-proteins, phospholipase C and InsP(3) receptors had no effect. These data suggest that PC1 and PC2 contribute to fluid-flow sensation by the primary cilium in renal epithelium and that they both function in the same mechanotransduction pathway. Loss or dysfunction of PC1 or PC2 may therefore lead to PKD owing to the inability of cells to sense mechanical cues that normally regulate tissue morphogenesis.
几种与多囊肾病(PKD)发病机制相关的蛋白质定位于纤毛。此外,在TgN737Rpw(编码北极星蛋白)发生突变的PKD小鼠中,纤毛形态异常。然而,尚不清楚在PKD中是否发生纤毛功能障碍或其与囊肿形成是否相关。在此,我们表明多囊蛋白-1(PC1)和多囊蛋白-2(PC2)分别由Pkd1和Pkd2编码,它们是人类常染色体显性PKD中突变基因的小鼠直系同源基因,在肾上皮细胞的初级纤毛中共分布。从缺乏功能性PC1的转基因小鼠中分离出的细胞形成了纤毛,但在生理流体流动刺激下不会增加Ca(2+)内流。针对PC2的阻断抗体同样消除了野生型细胞中的流动反应,ryanodine受体抑制剂也有同样的效果,而G蛋白、磷脂酶C和InsP(3)受体的抑制剂则没有作用。这些数据表明,PC1和PC2有助于肾上皮细胞初级纤毛感知流体流动,并且它们在同一机械转导途径中发挥作用。因此,PC1或PC2的缺失或功能障碍可能由于细胞无法感知正常调节组织形态发生的机械信号而导致PKD。