Vatta Matteo, Dumaine Robert, Antzelevitch Charles, Brugada Ramon, Li Hua, Bowles Neil E, Nademanee Koonlawee, Brugada Josep, Brugada Pedro, Towbin Jeffrey A
Department of Pediatrics (Cardiology), Baylor College of Medicine, Houston, TX, USA.
Mol Genet Metab. 2002 Apr;75(4):317-24. doi: 10.1016/S1096-7192(02)00006-9.
Brugada syndrome, an autosomal dominantly inherited form of ventricular fibrillation characterized by ST-segment elevation in leads V1-V3 and right bundle-branch block on surface electrocardiogram, is caused by mutations in the cardiac sodium channel gene SCN5A. Patients with Brugada syndrome were studied using single-strand conformation polymorphism analysis, denaturing high-performance liquid chromatography, and DNA sequencing of SCN5A. Mutations were identified in SCN5A in two families and one sporadic case. In one family, a missense mutation leading to a glycine to valine substitution (G351V) in the pore region between the DIS5 and DIS6 transmembrane segments was detected. Biophysical analysis demonstrated that this mutation caused significant current reduction. In the other family, a 20-bp deletion of the exon 5 splice acceptor site was identified; as exon 5 encodes part of the intracellular loop between DIS2 and DIS3, this portion of the channel is disrupted. In the sporadic patient, a missense mutation resulting in the substitution of lysine by glutamic acid (K126E) in the intracellular loop at the boundary with DIS1 was identified. These three new SCN5A mutations in Brugada syndrome patients are all located within domain I of SCN5A, a region not previously considered important in the development of ventricular arrhythmias.
Brugada综合征是一种常染色体显性遗传的心室颤动形式,其特征为体表心电图V1-V3导联ST段抬高及右束支传导阻滞,由心脏钠通道基因SCN5A突变引起。采用单链构象多态性分析、变性高效液相色谱法及SCN5A的DNA测序对Brugada综合征患者进行研究。在两个家族及1例散发病例中发现了SCN5A的突变。在一个家族中,检测到一个错义突变,导致在DIS5和DIS6跨膜片段之间的孔区域发生甘氨酸到缬氨酸的替换(G351V)。生物物理分析表明,该突变导致电流显著减少。在另一个家族中,发现外显子5剪接受体位点有20个碱基对的缺失;由于外显子5编码DIS2和DIS3之间细胞内环的一部分,通道的这一部分被破坏。在散发病例中,发现一个错义突变,导致在与DIS1边界处的细胞内环中赖氨酸被谷氨酸替换(K126E)。Brugada综合征患者中的这三个新的SCN5A突变均位于SCN5A的结构域I内,该区域以前在室性心律失常的发生中未被认为是重要的。