Birkenhäger R, Otto E, Schürmann M J, Vollmer M, Ruf E M, Maier-Lutz I, Beekmann F, Fekete A, Omran H, Feldmann D, Milford D V, Jeck N, Konrad M, Landau D, Knoers N V, Antignac C, Sudbrak R, Kispert A, Hildebrandt F
University Children's Hospital, Freiburg University, D-79106 Freiburg, Germany.
Nat Genet. 2001 Nov;29(3):310-4. doi: 10.1038/ng752.
Antenatal Bartter syndrome (aBS) comprises a heterogeneous group of autosomal recessive salt-losing nephropathies. Identification of three genes that code for renal transporters and channels as responsible for aBS has resulted in new insights into renal salt handling, diuretic action and blood-pressure regulation. A gene locus of a fourth variant of aBS called BSND, which in contrast to the other forms is associated with sensorineural deafness (SND) and renal failure, has been mapped to chromosome 1p. We report here the identification by positional cloning, in a region not covered by the human genome sequencing projects, of a new gene, BSND, as the cause of BSND. We examined ten families with BSND and detected seven different mutations in BSND that probably result in loss of function. In accordance with the phenotype, BSND is expressed in the thin limb and the thick ascending limb of the loop of Henle in the kidney and in the dark cells of the inner ear. The gene encodes a hitherto unknown protein with two putative transmembrane alpha-helices and thus might function as a regulator for ion-transport proteins involved in aBS, or else as a new transporter or channel itself.
产前巴特综合征(aBS)是一组异质性常染色体隐性失盐性肾病。已鉴定出三个编码肾转运体和通道的基因与aBS有关,这使人们对肾盐处理、利尿作用和血压调节有了新的认识。aBS的第四个变异型(称为BSND)的基因座已被定位到1号染色体,与其他类型不同,它与感音神经性耳聋(SND)和肾衰竭有关。我们在此报告,通过位置克隆在人类基因组测序项目未覆盖的区域鉴定出一个新基因BSND,它是BSND的病因。我们研究了10个患有BSND的家族,在BSND中检测到7种不同的突变,这些突变可能导致功能丧失。与该表型一致,BSND在肾髓袢升支细段和粗段以及内耳暗细胞中表达。该基因编码一种迄今未知的蛋白质,有两个假定的跨膜α螺旋,因此可能作为aBS相关离子转运蛋白的调节剂,或者本身作为一种新的转运体或通道发挥作用。