Department of Nephrology, Ruijin Hospital, School of Medicine, Shanghai Jiaotong University, Shanghai, China.
Clin Genet. 2010 Feb;77(2):155-62. doi: 10.1111/j.1399-0004.2009.01288.x. Epub 2009 Oct 6.
Mutations in the gene CLCNKB encoding the ClC-Kb chloride channel causes classic Bartter syndrome, which is characterized by hypokalaemic metabolic alkalosis, renal salt loss, hyper-reninaemic hyperaldosteronism and normal blood pressure. We aimed to investigate the underlying mutations in CLCNKB in two Chinese patients with classic Bartter syndrome and then test the effect of the mutations on ClC-Kb chloride channel activity. Mutation analysis of CLCNKB was performed by polymerase chain reaction (PCR) direct sequencing. Expression of the wild-type and mutant ClC-Kb was heterologous in Xenopus laevis oocytes. We identified three novel CLCNKB gene mutations, including one homozygous missense mutation (R351W) in one patient and two compound heterozygous mutations (R30X and A210V) in the other. As determined by two-electrode voltage-clamp analysis of ClC-Kb channel activity, R30X abolished the current amplitude; A210V and R351W significantly reduced the current amplitude. A210V was almost as sensitive as the wild type to extracellular pH and calcium, whereas R351W removed extracellular calcium activation and markedly reduced alkaline pH activation of ClC-Kb. The three novel CLCNKB mutations we identified in two Chinese patients with classic Bartter syndrome have a role in altering the functional properties of ClC-Kb channels.
基因突变导致 CLCNKB 编码的 ClC-Kb 氯离子通道,引起经典巴特综合征,其特征为低钾代谢性碱中毒、肾盐丢失、肾素-血管紧张素醛固酮升高和血压正常。我们旨在研究 2 例经典巴特综合征患者 CLCNKB 中潜在的突变,然后检测这些突变对 ClC-Kb 氯离子通道活性的影响。通过聚合酶链反应(PCR)直接测序进行 CLCNKB 突变分析。野生型和突变型 ClC-Kb 在非洲爪蟾卵母细胞中异源表达。我们鉴定了 3 种新的 CLCNKB 基因突变,包括 1 例患者的纯合错义突变(R351W)和另 1 例患者的两种复合杂合突变(R30X 和 A210V)。通过 ClC-Kb 通道活性的双电极电压钳分析,确定 R30X 消除了电流幅度;A210V 和 R351W 显著降低了电流幅度。A210V 对细胞外 pH 值和钙离子的敏感性几乎与野生型相同,而 R351W 消除了细胞外钙激活,并显著降低了 ClC-Kb 的碱性 pH 值激活。我们在 2 例中国经典巴特综合征患者中鉴定的 3 种新的 CLCNKB 突变改变了 ClC-Kb 通道的功能特性。