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芬兰 COFS 综合征患者中发现 ERCC6 启动子突变。

ERCC6 founder mutation identified in Finnish patients with COFS syndrome.

机构信息

Department of Clinical Genetics, Oulu University Hospital, University of Oulu, Oulu, Finland.

出版信息

Clin Genet. 2010 Dec;78(6):541-7. doi: 10.1111/j.1399-0004.2010.01424.x.

Abstract

Cerebro-oculo-facio-skeletal (COFS) syndrome is an autosomal recessive disorder characterized by microcephaly, congenital cataracts, facial dysmorphism, neurogenic arthrogryposis, growth failure and severe psychomotor retardation. We report a large consanguineous pedigree from northern Finland with six individuals belonging into four different sibships and affected with typical COFS syndrome phenotype. Two deceased patients have been published previously in 1982 as the first cases exhibiting cerebral calcifications typical for this disorder. Two living and one of the deceased patients were all shown to possess a novel homozygous mutation in the ERCC6 [Cockayne syndrome B (CSB)] gene, thereby confirming the diagnosis on molecular genetic level even for the earlier published cases. Genealogical investigation showed a common ancestor living in a northeastern village in Finland in the 18th century for all six patients implying a founder effect.

摘要

脑-眼-面-骨骼(COFS)综合征是一种常染色体隐性遗传病,其特征为小头畸形、先天性白内障、面部畸形、神经性关节强硬、生长发育迟缓以及严重的精神运动发育迟缓。我们报道了来自芬兰北部的一个大型近亲家族,该家族有 6 名成员,分属于 4 个不同的家系,具有典型的 COFS 综合征表型。其中 2 名已故患者曾于 1982 年首次报道,表现出该疾病特有的脑钙化。2 名在世患者和 1 名已故患者均携带 ERCC6 [Cockayne 综合征 B(CSB)]基因的新型纯合突变,因此即使对之前发表的病例也能在分子遗传学水平上明确诊断。家系调查显示,所有 6 名患者的共同祖先生活在 18 世纪芬兰东北部的一个村庄,提示存在一个奠基者效应。

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