Syrén Marie-Louise, Tedeschi Silvana, Cesareo Laila, Bellantuono Rosa, Colussi Giacomo, Procaccio Mirella, Alì Anna, Domenici Raffaele, Malberti Fabio, Sprocati Monica, Sacco Michele, Miglietti Nunzia, Edefonti Alberto, Sereni Fabio, Casari Giorgio, Coviello Domenico A, Bettinelli Alberto
Department of Pediatrics and Neonatology, University of Milan, Italy.
Hum Mutat. 2002 Jul;20(1):78. doi: 10.1002/humu.9045.
The SLC12A3 gene encodes the thiazide-sensitive Na-Cl co-transporter (NCCT) expressed in the apical membrane of the distal convoluted tubule of the kidney. Inactivating mutations of this gene are responsible for Gitelman syndrome (GS), a disorder inherited as an autosomal recessive trait. We searched for SLC12A3 gene mutations in 21 Italian patients with the clinical and biochemical features of GS (hypokalemia, hypomagnesemia, metabolic alkalosis, hypocalciuria, and the absence of nephrocalcinosis). All coding regions with their intron-exon boundaries were analyzed using PCR and SSCP techniques followed by sequencing analysis. We identified 21 different mutations evenly distributed throughout the gene without any mutation hot-spot. Fifteen are novel variants, including 12 missense mutations, one deletion, one deletion-insertion and one splice site mutation: R158Q, T163M, W172R, G316V, G374V, G463E, A464T, S615W, V677M, R852S, R958G, C985Y, 2114-2120delACCAAGT, 2144-2158delGCCTTCTACTCGGATinsTG, and 531-2A>G.
SLC12A3基因编码在肾远曲小管顶端膜中表达的噻嗪类敏感钠氯共转运体(NCCT)。该基因的失活突变导致吉特曼综合征(GS),这是一种常染色体隐性遗传疾病。我们在21名具有GS临床和生化特征(低钾血症、低镁血症、代谢性碱中毒、低钙尿症且无肾钙质沉着症)的意大利患者中寻找SLC12A3基因突变。使用PCR和SSCP技术并随后进行测序分析,对所有编码区及其内含子-外显子边界进行了分析。我们鉴定出21种不同的突变,这些突变均匀分布于整个基因,没有任何突变热点。其中15种是新的变异体,包括12种错义突变、1种缺失、1种缺失插入突变和1种剪接位点突变:R158Q、T163M、W172R、G316V、G374V、G463E、A464T、S615W、V677M、R852S、R958G、C985Y、2114 - 2120delACCAAGT、2144 - 2158delGCCTTCTACTCGGATinsTG和531 - 2A>G。