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一个患有多中心性骨质溶解伴结节病、关节炎及心脏缺陷的家族中发现一种新型基质金属蛋白酶2(MMP2)羧基端血红素结合蛋白结构域突变。

A novel matrix metalloproteinase 2 (MMP2) terminal hemopexin domain mutation in a family with multicentric osteolysis with nodulosis and arthritis with cardiac defects.

作者信息

Tuysuz Beyhan, Mosig Rebecca, Altun Gürkan, Sancak Selim, Glucksman Marc J, Martignetti John A

机构信息

Department of Pediatrics, Division of Genetics,Istanbul University Cerrahpasa Faculty of Medicine, Istanbul, Turkey.

出版信息

Eur J Hum Genet. 2009 May;17(5):565-72. doi: 10.1038/ejhg.2008.204. Epub 2008 Nov 5.

Abstract

Multicentric osteolysis with nodulosis and arthropathy (MONA, NAO (OMIM no. 605156)) is an autosomal recessive member of the 'vanishing bone' syndromes and is notable for the extent of carpal and tarsal osteolysis and interphalangeal joint erosions, facial dysmorphia, and the presence of fibrocollagenous nodules. This rare disorder has been described previously in Saudi Arabian and Indian families. We now report on the first Turkish family with MONA, further confirming the panethnic nature of this disease. Strikingly, and in addition to the previously noted skeletal and joint features, affected members of this family also had congenital heart defects. Molecular analysis identified a novel MMP2 inactivating mutation that deletes the terminal hemopexin domains and thus confirmed the diagnosis of MONA. On the basis of these findings, we suggest that cardiac defects may also represent a component of this syndrome and thus a physiologically relevant target of MMP-2 activity.

摘要

多中心性骨质溶解伴结节病和关节病(MONA,NAO,OMIM编号605156)是“骨质消失”综合征中的常染色体隐性遗传病,以腕骨和跗骨骨质溶解程度、指间关节侵蚀、面部畸形以及纤维胶原性结节的存在为显著特征。这种罕见疾病此前已在沙特阿拉伯和印度家族中有过描述。我们现在报告首例患有MONA的土耳其家族,进一步证实了该病的全种族特性。令人惊讶的是,除了先前指出的骨骼和关节特征外,该家族的患病成员还患有先天性心脏缺陷。分子分析鉴定出一种新的MMP2失活突变,该突变缺失了末端血红素结合蛋白结构域,从而确诊为MONA。基于这些发现,我们认为心脏缺陷可能也是该综合征的一个组成部分,因此是MMP - 2活性的一个生理相关靶点。

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