Institute of Developmental Biology, University of Cologne, Cologne, Germany; Center for Molecular Medicine Cologne, Medical Faculty, University of Cologne, Cologne, Germany.
Center for Biochemistry, University of Cologne, Cologne, Germany.
J Invest Dermatol. 2014 May;134(5):1313-1322. doi: 10.1038/jid.2013.492. Epub 2013 Nov 14.
Fraser syndrome (FS) is a phenotypically variable, autosomal recessive disorder characterized by cryptophthalmus, cutaneous syndactyly, and other malformations resulting from mutations in FRAS1, FREM2, and GRIP1. Transient embryonic epidermal blistering causes the characteristic defects of the disorder. Fras1, Frem1, and Frem2 form the extracellular Fraser complex, which is believed to stabilize the basement membrane. However, several cases of FS could not be attributed to mutations in FRAS1, FREM2, or GRIP1, and FS displays high clinical variability, suggesting that there is an additional genetic, possibly modifying contribution to this disorder. An extracellular matrix protein containing VWA-like domains related to those in matrilins and collagens (AMACO), encoded by the VWA2 gene, has a very similar tissue distribution to the Fraser complex proteins in both mouse and zebrafish. Here, we show that AMACO deposition is lost in Fras1-deficient zebrafish and mice and that Fras1 and AMACO interact directly via their chondroitin sulfate proteoglycan (CSPG) and P2 domains. Knockdown of vwa2, which alone causes no phenotype, enhances the phenotype of hypomorphic Fras1 mutant zebrafish. Together, our data suggest that AMACO represents a member of the Fraser complex.
弗雷泽综合征(FS)是一种表型多变的常染色体隐性疾病,其特征为隐眼、皮肤并指畸形和其他由 FRAS1、FREM2 和 GRIP1 突变引起的畸形。胚胎期表皮暂时性水疱导致了该疾病的特征性缺陷。Fras1、Frem1 和 Frem2 形成细胞外 Fraser 复合物,据信该复合物稳定基底膜。然而,有几例 FS 不能归因于 FRAS1、FREM2 或 GRIP1 的突变,并且 FS 表现出高度的临床变异性,这表明该疾病存在其他遗传因素,可能是修饰基因的作用。含有类似于 matrilins 和胶原的 VWA 样结构域的细胞外基质蛋白(AMACO),由 VWA2 基因编码,在小鼠和斑马鱼中与 Fraser 复合物蛋白具有非常相似的组织分布。在这里,我们表明 Fras1 缺陷的斑马鱼和小鼠中 AMACO 的沉积丢失,并且 Fras1 和 AMACO 通过它们的软骨素硫酸盐蛋白聚糖(CSPG)和 P2 结构域直接相互作用。单独引起无表型的 vwa2 的敲低增强了 Fras1 突变体斑马鱼的低表型。综上所述,我们的数据表明 AMACO 是 Fraser 复合物的一个成员。