X连锁显性肩胛腓骨肌病是由编码四又二分之一LIM结构域蛋白1的基因突变所致。

X-linked dominant scapuloperoneal myopathy is due to a mutation in the gene encoding four-and-a-half-LIM protein 1.

作者信息

Quinzii Catarina M, Vu Tuan H, Min K Christopher, Tanji Kurenai, Barral Sandra, Grewal Raji P, Kattah Andrea, Camaño Pilir, Otaegui David, Kunimatsu Teruhito, Blake David M, Wilhelmsen Kirk C, Rowland Lewis P, Hays Arthur P, Bonilla Eduardo, Hirano Michio

机构信息

Department of Neurology, Columbia University Medical Center, New York, NY 10032, USA.

出版信息

Am J Hum Genet. 2008 Jan;82(1):208-13. doi: 10.1016/j.ajhg.2007.09.013.

Abstract

Scapuloperoneal (SP) syndrome encompasses heterogeneous neuromuscular disorders characterized by weakness in the shoulder-girdle and peroneal muscles. In a large Italian-American pedigree with dominant SP myopathy (SPM) previously linked to chromosome 12q, we have mapped the disease to Xq26, and, in all of the affected individuals, we identified a missense change (c.365G-->C) in the FHL1 gene encoding four-and-a-half-LIM protein 1 (FHL1). The mutation substitutes a serine for a conserved trypophan at amino acid 122 in the second LIM domain of the protein. Western blot analyses of muscle extracts revealed FHL1 loss that paralleled disease severity. FHL1 and an isoform, FHL1C, are highly expressed in skeletal muscle and may contribute to stability of sarcomeres and sarcolemma, myofibrillary assembly, and transcriptional regulation. This is the first report, to our knowledge, of X-linked dominant SP myopathy and the first human mutation in FHL1.

摘要

肩胛腓骨(SP)综合征涵盖了多种异质性神经肌肉疾病,其特征为肩胛带肌和腓骨肌无力。在一个与12号染色体相关的显性SP肌病(SPM)的意大利裔美大家族中,我们将该疾病定位到了Xq26,并且在所有受影响个体中,我们在编码四又二分之一LIM结构域蛋白1(FHL1)的FHL1基因中鉴定出一个错义变化(c.365G→C)。该突变使蛋白质第二个LIM结构域中第122位氨基酸的保守色氨酸被丝氨酸替代。对肌肉提取物的蛋白质免疫印迹分析显示,FHL1的缺失与疾病严重程度平行。FHL1及其异构体FHL1C在骨骼肌中高度表达,可能有助于肌节和肌膜的稳定性、肌原纤维组装以及转录调控。据我们所知,这是关于X连锁显性SP肌病的首次报道,也是FHL1中的首例人类突变。

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