Patricia Tomás-Simó, Antonio Sierra-Rivera, Ana Checa-Ros, Raquel Rodríguez-López, Antonio Galán-Serrano, Luis D'Marco
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A next-generation sequencing (NGS) test was performed on her and her child (no other relatives alive), who was also asymptomatic with no obvious electrolytic abnormalities. Two mutations confirmed as pathogenic were found in the SLC12A3 gene (NM_000339.3) of the mother, c.704C>G and c.704C>T, as well as a new heterozygous variant in trans not reported before, c.704C>A p.(Thr235Lys), and identified as a variant of uncertain significance (VUS). This new VUS (c.704C>A p) was also present in the child, increasing evidence of its potential pathogenicity. The new SLC12A3 gene variant could represent a pathogenic mutation associated with GS. The use of NGS-based panel is recommended to cover the large genotypic variability associated with this disease, in an attempt to identify novel SLC12A3 gene variants of potential pathogenicity.</p>","PeriodicalId":520716,"journal":{"name":"Nephrology (Carlton, Vic.)","volume":"30 9","pages":"e70127"},"PeriodicalIF":1.9000,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12433184/pdf/","citationCount":"0","resultStr":"{\"title\":\"Novel Pathogenic Genotype in SLC12A3 Associated to Gitelman Syndrome: A Case Report.\",\"authors\":\"Patricia Tomás-Simó, Antonio Sierra-Rivera, Ana Checa-Ros, Raquel Rodríguez-López, Antonio Galán-Serrano, Luis D'Marco\",\"doi\":\"10.1111/nep.70127\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Gitelman syndrome (GS) is considered one of the most common hereditary renal tubular disorders, characterised by hypokalemia, hypomagnesemia, hypocalciuria, and metabolic alkalosis. The primary cause of this disorder resides in the SLC12A3 gene, which encodes the NaCl cotransporter in the distal convoluted tubule, and for which more than 500 mutations associated with GS have been described. We present the case of a 51-year-old female referred for evaluation of recurrent hypokalemia and hypomagnesemia, with no clinical symptoms. The blood test also revealed hypocalciuria and metabolic alkalosis. Oral supplementation with potassium and magnesium was prescribed. A next-generation sequencing (NGS) test was performed on her and her child (no other relatives alive), who was also asymptomatic with no obvious electrolytic abnormalities. Two mutations confirmed as pathogenic were found in the SLC12A3 gene (NM_000339.3) of the mother, c.704C>G and c.704C>T, as well as a new heterozygous variant in trans not reported before, c.704C>A p.(Thr235Lys), and identified as a variant of uncertain significance (VUS). This new VUS (c.704C>A p) was also present in the child, increasing evidence of its potential pathogenicity. The new SLC12A3 gene variant could represent a pathogenic mutation associated with GS. 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引用次数: 0
摘要
Gitelman综合征(GS)被认为是最常见的遗传性肾小管疾病之一,以低钾血症、低镁血症、低钙尿和代谢性碱中毒为特征。这种疾病的主要原因在于SLC12A3基因,该基因编码远曲小管中的NaCl共转运体,已有超过500种与GS相关的突变被描述。我们提出的情况下,51岁的女性转介评估复发性低钾血症和低镁血症,没有临床症状。血液检查还显示低钙尿和代谢性碱中毒。处方口服补充钾和镁。对她和她的孩子(没有其他亲属在世)进行了下一代测序(NGS)测试,她的孩子也无症状,没有明显的电解质异常。在母亲SLC12A3基因(NM_000339.3) c.704C b> G和c.704C>T中发现了两个致病突变,在trans中发现了一个新的未报道的杂合变异c.704C> a p.(Thr235Lys),并鉴定为不确定意义变异(VUS)。这种新的VUS (c.704C . > . A . p)也存在于儿童中,增加了其潜在致病性的证据。新的SLC12A3基因变异可能代表与GS相关的致病突变。建议使用基于ngs的面板来覆盖与该疾病相关的大基因型变异性,以试图确定具有潜在致病性的新型SLC12A3基因变异。
Novel Pathogenic Genotype in SLC12A3 Associated to Gitelman Syndrome: A Case Report.
Gitelman syndrome (GS) is considered one of the most common hereditary renal tubular disorders, characterised by hypokalemia, hypomagnesemia, hypocalciuria, and metabolic alkalosis. The primary cause of this disorder resides in the SLC12A3 gene, which encodes the NaCl cotransporter in the distal convoluted tubule, and for which more than 500 mutations associated with GS have been described. We present the case of a 51-year-old female referred for evaluation of recurrent hypokalemia and hypomagnesemia, with no clinical symptoms. The blood test also revealed hypocalciuria and metabolic alkalosis. Oral supplementation with potassium and magnesium was prescribed. A next-generation sequencing (NGS) test was performed on her and her child (no other relatives alive), who was also asymptomatic with no obvious electrolytic abnormalities. Two mutations confirmed as pathogenic were found in the SLC12A3 gene (NM_000339.3) of the mother, c.704C>G and c.704C>T, as well as a new heterozygous variant in trans not reported before, c.704C>A p.(Thr235Lys), and identified as a variant of uncertain significance (VUS). This new VUS (c.704C>A p) was also present in the child, increasing evidence of its potential pathogenicity. The new SLC12A3 gene variant could represent a pathogenic mutation associated with GS. The use of NGS-based panel is recommended to cover the large genotypic variability associated with this disease, in an attempt to identify novel SLC12A3 gene variants of potential pathogenicity.