Department of Biomedical Sciences, Gabriele d'Annunzio University, Chieti, Italy.
Am J Hum Genet. 2010 Aug 13;87(2):265-73. doi: 10.1016/j.ajhg.2010.07.003.
Ectodermal dysplasias form a large disease family with more than 200 members. The combination of hair and tooth abnormalities, alopecia, and cutaneous syndactyly is characteristic of ectodermal dysplasia-syndactyly syndrome (EDSS). We used a homozygosity mapping approach to map the EDSS locus to 1q23 in a consanguineous Algerian family. By candidate gene analysis, we identified a homozygous mutation in the PVRL4 gene that not only evoked an amino acid change but also led to exon skipping. In an Italian family with two siblings affected by EDSS, we further detected a missense and a frameshift mutation. PVRL4 encodes for nectin-4, a cell adhesion molecule mainly implicated in the formation of cadherin-based adherens junctions. We demonstrated high nectin-4 expression in hair follicle structures, as well as in the separating digits of murine embryos, the tissues mainly affected by the EDSS phenotype. In patient keratinocytes, mutated nectin-4 lost its capability to bind nectin-1. Additionally, in discrete structures of the hair follicle, we found alterations of the membrane localization of nectin-afadin and cadherin-catenin complexes, which are essential for adherens junction formation, and we found reorganization of actin cytoskeleton. Together with cleft lip and/or palate ectodermal dysplasia (CLPED1, or Zlotogora-Ogur syndrome) due to an impaired function of nectin-1, EDSS is the second known "nectinopathy" caused by mutations in a nectin adhesion molecule.
外胚层发育不全症是一个拥有超过 200 个成员的大型疾病家族。毛发和牙齿异常、脱发和皮肤并指畸形的组合是外胚层发育不全并指综合征(EDSS)的特征。我们使用纯合子作图方法将 EDSS 基因座定位到一个近亲阿尔及利亚家庭的 1q23。通过候选基因分析,我们在一个受影响的同胞姐弟的意大利家庭中发现了 PVRL4 基因的纯合突变,该突变不仅引起了氨基酸改变,还导致外显子跳跃。PVRL4 基因编码黏附分子 nectin-4,它主要参与钙黏蛋白黏附连接的形成。我们证明了高 nectin-4 表达存在于毛囊结构以及鼠胚胎的分离指中,这些组织主要受 EDSS 表型影响。在患者角质形成细胞中,突变的 nectin-4 丧失了与 nectin-1 结合的能力。此外,在毛囊的离散结构中,我们发现 nectin-afadin 和钙黏蛋白-catenin 复合物的膜定位发生改变,这对于黏附连接的形成至关重要,并且我们还发现肌动蛋白细胞骨架的重组。与由于 nectin-1 功能障碍引起的唇裂和/或腭裂外胚层发育不全症(CLPED1,或 Zlotogora-Ogur 综合征)一起,EDSS 是由黏附分子 nectin 突变引起的第二种已知的“nectinopathy”。
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