Simon D B, Karet F E, Hamdan J M, DiPietro A, Sanjad S A, Lifton R P
Howard Hughes Medical Institute, Department of Medicine, Boyer Center for Molecular Medicine, Yale University School of Medicine, New Haven, Connecticut 06510, USA.
Nat Genet. 1996 Jun;13(2):183-8. doi: 10.1038/ng0696-183.
Inherited hypokalaemic alkalosis with low blood pressure can be divided into two groups-Gitelman's syndrome, featuring hypocalciuria, hypomagnesaemia and milder clinical manifestations, and Bartter's syndrome, featuring hypercalciuria and early presentation with severe volume depletion. Mutations in the renal Na-Cl cotransporter have been shown to cause Gitelman's syndrome. We demonstrate linkage of Bartter's syndrome to the renal Na-K-2Cl cotransporter gene NKCC2, and identify frameshift or non-conservative missense mutations for this gene that co-segregate with the disease. These findings demonstrate the molecular basis of Bartter's syndrome, provide the basis for molecular classification of patients with inherited hypokalaemic alkalosis, and suggest potential phenotypes in heterozygous carriers of NKCC2 mutations.
吉特曼综合征,其特征为低钙尿症、低镁血症及较轻的临床表现;巴特综合征,其特征为高钙尿症及早期出现严重的容量耗竭。已证实肾钠 - 氯共转运体的突变可导致吉特曼综合征。我们证明了巴特综合征与肾钠 - 钾 - 2氯共转运体基因NKCC2连锁,并鉴定出该基因的移码突变或非保守错义突变,这些突变与疾病共分离。这些发现揭示了巴特综合征的分子基础,为遗传性低钾性碱中毒患者的分子分类提供了依据,并提示了NKCC2突变杂合携带者的潜在表型。