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Ehlers-Danlos/肌病重叠综合征由 COL12A1 中的大片从头缺失引起。

Ehlers-Danlos/myopathy overlap syndrome caused by a large de novo deletion in COL12A1.

机构信息

ULB Center of Human Genetics, Hôpital Erasme, Brussels, Belgium.

Institute for Dental Research and Oral Musculoskeletal Biology and Center for Biochemistry, Faculty of Medicine and University Hospital Cologne, University of Cologne, Köln, Germany.

出版信息

Am J Med Genet A. 2022 May;188(5):1556-1561. doi: 10.1002/ajmg.a.62653. Epub 2022 Jan 12.

Abstract

Autosomal dominant and recessive mutations in COL12A1 cause the Ehlers-Danlos/myopathy overlap syndrome. Here, we describe a boy with fetal hypokinesia, severe neonatal weakness, striking hyperlaxity, high arched palate, retrognathia, club feet, and pectus excavatum. His motor development was initially delayed but muscle strength improved with time while hyperlaxity remained very severe causing recurrent joint dislocations. Using trio exome sequencing and a copy number variation (CNV) analysis tool, we identified an in-frame de novo heterozygous deletion of the exons 45 to 54 in the COL12A1 gene. Collagen XII immunostaining on cultured skin fibroblasts demonstrated intracellular retention of collagen XII, supporting the pathogenicity of the deletion. The phenotype of our patient is slightly more severe than other cases with dominantly acting mutations, notably with the presence of fetal hypokinesia. This case highlights the importance of CNVs analysis in the COL12A1 gene in patients with a phenotype suggesting Ehlers-Danlos/myopathy overlap syndrome.

摘要

COL12A1 上的常染色体显性和隐性突变可导致 Ehlers-Danlos/肌病重叠综合征。在这里,我们描述了一名患有胎儿运动减少、严重新生儿肌无力、明显过度伸展、高腭弓、小下颌、马蹄足和漏斗胸的男孩。他的运动发育最初延迟,但随着时间的推移,肌肉力量逐渐增强,而过度伸展仍然非常严重,导致反复关节脱位。通过三人体外显子组测序和拷贝数变异 (CNV) 分析工具,我们在 COL12A1 基因中发现了一个外显子 45 至 54 的无义杂合缺失。对培养的皮肤成纤维细胞进行的 XII 型胶原免疫染色显示,胶原 XII 在内质网中滞留,支持该缺失的致病性。我们患者的表型比其他具有显性突变的病例略严重,特别是存在胎儿运动减少。该病例强调了在 COL12A1 基因中进行 CNV 分析在提示 Ehlers-Danlos/肌病重叠综合征的患者中的重要性。

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