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核纤层蛋白A/C基因频繁的低外显率突变,导致埃默里-德赖富斯肌营养不良症。

Frequent low penetrance mutations in the Lamin A/C gene, causing Emery Dreifuss muscular dystrophy.

作者信息

Vytopil Michal, Ricci Enzo, Dello Russo Antonio, Hanisch Frank, Neudecker Stephan, Zierz Stephan, Ricotti Roberta, Demay Laurence, Richard Pascale, Wehnert Manfred, Bonne Gisèle, Merlini Luciano, Toniolo Daniela

机构信息

Institute of Molecular Genetics-CNR, Via Abbiategrasso, 207, 27100, Pavia, Italy.

出版信息

Neuromuscul Disord. 2002 Dec;12(10):958-63. doi: 10.1016/s0960-8966(02)00178-5.

Abstract

Emery Dreifuss muscular dystrophy is a genetically heterogeneous disorder characterized by the clinical triad of early onset contractures, progressive muscular wasting and weakness with humeroperoneal distribution and cardiac conduction defects. Mutations in the Lamin A/C (LMNA) gene are responsible for the autosomal dominant and the autosomal recessive forms. Familiar and sporadic patients carrying mutations in the LMNA gene show high variability in the clinical symptomatology and age of onset. In this report, we describe four families harboring missense mutations in the LMNA gene and we show that the effect of mutations ranges from silent to fully penetrant. We suggest that incomplete penetrance of dominant mutations in the LMNA gene is a common feature and we emphasize the significance of mutational analysis in relatives of sporadic cases of laminopathies, as asymptomatic carriers face high risk of sudden cardiac death.

摘要

埃默里-德赖富斯肌营养不良症是一种基因异质性疾病,其临床特征为三联征:早发性挛缩、进行性肌肉萎缩和无力,呈肱腓型分布以及心脏传导缺陷。核纤层蛋白A/C(LMNA)基因突变导致常染色体显性和常染色体隐性形式。携带LMNA基因突变的家族性和散发性患者在临床症状和发病年龄上表现出高度变异性。在本报告中,我们描述了四个携带LMNA基因错义突变的家族,并表明突变的影响范围从无明显影响到完全显性。我们认为LMNA基因显性突变的不完全显性是一个常见特征,并强调在散发性核纤层蛋白病病例的亲属中进行突变分析的重要性,因为无症状携带者面临心脏性猝死的高风险。

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