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患有贝克尔型肌强直的摩洛哥近亲家族及文献综述

Moroccan consanguineous family with Becker myotonia and review.

作者信息

Ratbi Ilham, Elalaoui Siham Chafai, Escudero Adela, Kriouile Yamina, Molano Jesus, Sefiani Abdelaziz

机构信息

Human Genomic Center, Faculty of medicine and pharmacy, University Mohammed V Souissi, Rabat, Morocco.

出版信息

Ann Indian Acad Neurol. 2011 Oct;14(4):307-9. doi: 10.4103/0972-2327.91963.

Abstract

Myotonia congenita is a genetic muscle disorder characterized by clinical and electrical myotonia, muscle hypertrophy, and stiffness. It is inherited as either autosomal-dominant or -recessive, known as Thomsen and Becker diseases, respectively. These diseases are distinguished by the severity of their symptoms and their patterns of inheritance. Becker disease usually appears later in childhood than Thomsen disease and causes more severe muscle stiffness and pain. Mutations in the muscular voltage-dependent chloride channel gene (CLCN1), located at 7q35, have been found in both types. We report here the case of a Moroccan consanguineous family with a myotonic autosomal-recessive condition in two children. The molecular studies showed that the patients reported here are homozygous for mutation p.Gly482Arg in the CLCN1 gene. The parents were heterozygote carriers for mutation p.Gly482Arg. This diagnosis allowed us to provide an appropriate management to the patients and to make a genetic counselling to their family.

摘要

先天性肌强直是一种遗传性肌肉疾病,其特征为临床和电生理方面的肌强直、肌肉肥大及僵硬。它以常染色体显性或隐性方式遗传,分别被称为汤姆森病和贝克尔病。这两种疾病通过症状严重程度和遗传模式加以区分。贝克尔病通常比汤姆森病在儿童期出现得更晚,且会导致更严重的肌肉僵硬和疼痛。两种类型均发现位于7q35的肌肉电压依赖性氯通道基因(CLCN1)发生了突变。我们在此报告一个摩洛哥近亲家庭的病例,该家庭中有两名儿童患有常染色体隐性遗传性肌强直病症。分子研究表明,此处报告的患者在CLCN1基因中为p.Gly482Arg突变的纯合子。父母是p.Gly482Arg突变的杂合子携带者。这一诊断使我们能够为患者提供适当的治疗,并为其家人进行遗传咨询。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1cf9/3271475/f412c51fb629/AIAN-14-307-g002.jpg

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