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中央轴空病的一种隐性形式,短暂表现为多微小轴空病,与1型兰尼碱受体基因的纯合突变有关。

A recessive form of central core disease, transiently presenting as multi-minicore disease, is associated with a homozygous mutation in the ryanodine receptor type 1 gene.

作者信息

Ferreiro Ana, Monnier Nicole, Romero Norma B, Leroy Jean-Paul, Bönnemann Carsten, Haenggeli Charles-Antoine, Straub Volker, Voss Wolfgang D, Nivoche Yves, Jungbluth Heinz, Lemainque Arnaud, Voit Thomas, Lunardi Joël, Fardeau Michel, Guicheney Pascale

机构信息

INSERM U523, Institut de Myologie, Groupe Hospitalier Pitié-Salpêtrière, 47 boulevard de l'Hôpital, 75651 Paris, France.

出版信息

Ann Neurol. 2002 Jun;51(6):750-9. doi: 10.1002/ana.10231.

Abstract

Multi-minicore disease is an autosomal recessive congenital myopathy characterized by the presence of multiple, short-length core lesions (minicores) in both muscle fiber types. These lesions being nonspecific and the clinical phenotype being heterogeneous, multi-minicore disease boundaries remain unclear. To identify its genetic basis, we performed a genome-wide screening in a consanguineous Algerian family in which three children presented in infancy with moderate weakness predominant in axial muscles, pelvic girdle and hands, joint hyperlaxity (hand involvement phenotype), and multiple minicores. We mapped the disease to chromosome 19q13 in this family and, subsequently, in three additional families showing a similar phenotype, with a maximum LOD score of 5.19 for D19S570. This locus was excluded in 16 other multi-minicore disease families with predominantly axial weakness, scoliosis, and respiratory insufficiency ("classical" phenotype). In the Algerian family, we identified a novel homozygous missense mutation (P3527S) in the ryanodine receptor type 1 gene, a positional candidate gene responsible for the autosomal dominant congenital myopathy central core disease. New muscle biopsies from the three patients at adulthood demonstrated typical central core disease with rods; no cores were found in the healthy parents. This subgroup of families linked to 19q13 represents the first variant of central core disease with genetically proven recessive inheritance and transient presentation as multi-minicore disease.

摘要

多微小核病是一种常染色体隐性先天性肌病,其特征是在两种肌纤维类型中均存在多个短长度的核心病变(微小核)。这些病变不具有特异性,临床表型具有异质性,多微小核病的界限仍不明确。为了确定其遗传基础,我们对一个近亲结婚的阿尔及利亚家庭进行了全基因组筛查,该家庭中有三个孩子在婴儿期出现中度肌无力,主要累及躯干肌、骨盆带肌和手部,关节过度松弛(手部受累表型)以及多个微小核。我们将该疾病定位到这个家庭的19号染色体长臂1区3带,随后又在另外三个表现出相似表型的家庭中进行了定位,D19S570的最大对数优势分数为5.19。在另外16个以躯干肌无力、脊柱侧弯和呼吸功能不全为主的多微小核病家庭(“经典”表型)中排除了该基因座。在这个阿尔及利亚家庭中,我们在1型兰尼碱受体基因中发现了一个新的纯合错义突变(P3527S),该基因是导致常染色体显性先天性肌病中央核心病的一个定位候选基因。这三名患者成年后的新肌肉活检显示出典型的伴有杆状体的中央核心病;在健康父母中未发现核心病变。与19q13相关的这一亚组家庭代表了中央核心病的首个变异类型,其遗传方式经证实为隐性遗传,并曾短暂表现为多微小核病。

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