离子通道病
先天性肌强直
桑格测序
基因型
遗传学
先证者
生物
肌强直
基因
分子生物学
医学
内科学
突变
强直性营养不良
作者
Nikolaos M. Marinakis,Maria Svingou,Giorgos‐Konstantinos Papadimas,Constantinos Papadopoulos,Elisabeth Chroni,Roser Pons,Evangelos Pavlou,Ioannis Sarmas,Konstantina Kosma,Paraskevi Apostolou,Christalena Sofocleous,Joanne Traeger‐Synodinos,Kyriaki Kekou
摘要
Myotonia congenita (MC) is the most common hereditary channelopathy in humans. Characterized by muscle stiffness, MC may be transmitted as either an autosomal dominant (Thomsen) or a recessive (Becker) disorder. MC is caused by variants in the voltage-gated chloride channel 1 (CLCN1) gene, important for the normal repolarization of the muscle action potential. More than 250 disease-causing variants in the CLCN1 gene have been reported. This study provides an MC genotype-phenotype spectrum in a large cohort of Greek patients and focuses on novel variants and disease epidemiology, including additional insights for the variant CLCN1:c.501C > G.
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