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一个新的 KCNA1 突变导致抽搐和低镁血症。

A de novo KCNA1 Mutation in a Patient with Tetany and Hypomagnesemia.

机构信息

Department of Physiology, Radboud Institute for Molecular Life Sciences, Radboud University Medical Center, Nijmegen, the Netherlands.

Department of General Pediatrics, University Children's Hospital, Münster, Germany.

出版信息

Nephron. 2018;139(4):359-366. doi: 10.1159/000488954. Epub 2018 May 23.

Abstract

Mutations in the KCNA1 gene encoding the voltage-gated potassium (K+) channel Kv1.1 have been linked to rare neurological syndromes, episodic ataxia type 1 (EA1) and myokymia. In 2009, a KCNA1 mutation was identified in a large family with autosomal dominant hypomagnesemia. Despite efforts in establishing a genotype-phenotype correlation for the wide variety of symptoms in EA1, little is known on the serum magnesium (Mg2+) levels in these patients. In the present study, we describe a new de novo KCNA1 mutation in a Polish patient with tetany and hypomagnesemia. Electrophysiological and biochemical analyses were performed to determine the pathogenicity of the mutation. A female patient presented with low serum Mg2+ levels, renal Mg2+ wasting, muscle cramps, and tetanic episodes. Whole exome sequencing identified a p.Leu328Val mutation in KCNA1 encoding the Kv1.1 K+ channel. Electrophysiological examinations demonstrated that the p.Leu328Val mutation caused a dominant-negative loss of function of the encoded Kv1.1 channel. Cell surface biotinylation showed normal plasma membrane expression. Taken together, this is the second report linking KCNA1 with hypomagnesemia, thereby emphasizing the need for further evaluation of the clinical phenotypes observed in patients carrying KCNA1 mutations.

摘要

KCNA1 基因突变与电压门控钾 (K+) 通道 Kv1.1 相关,可导致罕见的神经综合征,包括发作性共济失调 1 型 (EA1) 和肌束颤动。2009 年,在一个常染色体显性低镁血症的大家族中发现了 KCNA1 突变。尽管人们努力为 EA1 的各种症状建立基因型-表型相关性,但对于这些患者的血清镁 (Mg2+) 水平知之甚少。在本研究中,我们描述了一名波兰患者的新的从头 KCNA1 突变,该患者患有抽搐和低镁血症。进行了电生理和生化分析以确定突变的致病性。一名女性患者出现低血清 Mg2+ 水平、肾镁丢失、肌肉痉挛和抽搐发作。全外显子组测序鉴定出 KCNA1 中编码 Kv1.1 K+ 通道的 p.Leu328Val 突变。电生理检查表明,p.Leu328Val 突变导致编码的 Kv1.1 通道的显性负性失活。细胞表面生物素化显示正常的质膜表达。总之,这是第二个将 KCNA1 与低镁血症联系起来的报告,从而强调需要进一步评估携带 KCNA1 突变的患者的临床表型。

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