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I型胶原蛋白的α2(I)链完全缺失会导致一种罕见的埃勒斯-当洛综合征,其特征为关节活动过度以及易患心脏瓣膜问题。

Total absence of the alpha2(I) chain of collagen type I causes a rare form of Ehlers-Danlos syndrome with hypermobility and propensity to cardiac valvular problems.

作者信息

Malfait F, Symoens S, Coucke P, Nunes L, De Almeida S, De Paepe A

出版信息

J Med Genet. 2006 Jul;43(7):e36. doi: 10.1136/jmg.2005.038224.

Abstract

BACKGROUND

Heterozygous mutations in the COL1A1 or COL1A2 gene encoding the alpha1 and alpha2 chain of type I collagen generally cause either osteogenesis imperfecta or the arthrochalasis form of Ehlers-Danlos syndrome (EDS). Homozygous or compound heterozygous COL1A2 mutations resulting in complete deficiency of the proalpha2(I) collagen chains are extremely rare and have been reported in only a few patients, albeit with variable phenotypic outcome.

METHODS

The clinical features of the proband, a 6 year old boy, were recorded. Analysis of proalpha and alpha-collagen chains was performed by SDS-polyacrylamide gel electrophoresis using the Laemmli buffer system. Single stranded conformation polymorphism analysis of the proband's DNA was also carried out.

RESULTS

In this report we show that complete lack of proalpha2(I) collagen chains can present as a phenotype reminiscent of mild hypermobility EDS during childhood.

CONCLUSIONS

Biochemical analysis of collagens extracted from skin fibroblasts is a powerful tool to detect the subset of patients with complete absence of proalpha2(I) collagen chains, and in these patients, careful cardiac follow up with ultrasonography is highly recommended because of the risk for cardiac valvular problems in adulthood.

摘要

背景

编码I型胶原蛋白α1和α2链的COL1A1或COL1A2基因的杂合突变通常会导致成骨不全或埃勒斯-当洛综合征(EDS)的关节松弛型。导致前α2(I)胶原链完全缺乏的纯合或复合杂合COL1A2突变极为罕见,仅在少数患者中报道过,尽管其表型结果各异。

方法

记录了先证者(一名6岁男孩)的临床特征。使用Laemmli缓冲系统通过SDS-聚丙烯酰胺凝胶电泳对前α和α胶原链进行分析。还对先证者的DNA进行了单链构象多态性分析。

结果

在本报告中,我们表明前α2(I)胶原链的完全缺乏在儿童期可表现为类似于轻度关节过度活动型EDS的表型。

结论

对从皮肤成纤维细胞中提取的胶原蛋白进行生化分析是检测前α2(I)胶原链完全缺乏的患者亚组的有力工具,对于这些患者,由于成年后存在心脏瓣膜问题的风险,强烈建议通过超声心动图进行仔细的心脏随访。

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