Kutkowska-Kaźmierczak Anna, Niepokój Katarzyna, Wertheim-Tysarowska Katarzyna, Giza Aleksandra, Mordasewicz-Goliszewska Maria, Bal Jerzy, Obersztyn Ewa
Department of Medical Genetics, The Institute of Mother and Child, ul.Kasprzaka 17a, 01-211, Warsaw, Poland,
J Appl Genet. 2015 Aug;56(3):329-37. doi: 10.1007/s13353-014-0266-1. Epub 2015 Jan 10.
Connexins belong to the family of gap junction proteins which enable direct cell-to-cell communication by forming channels in adjacent cells. Mutations in connexin genes cause a variety of human diseases and, in a few cases, result in skin disorders. There are significant differences in the clinical picture of two rare autosomal dominant syndromes: keratitis-ichthyosis-deafness (KID) syndrome and hidrotic ectodermal dysplasia (Clouston syndrome), which are caused by GJB2 and GJB6 mutations, respectively. This is despite the fact that, in both cases, malfunctioning of the same family proteins and some overlapping clinical features (nail dystrophy, hair loss, and palmoplantar keratoderma) is observed. KID syndrome is characterized by progressive vascularizing keratitis, ichthyosiform erythrokeratoderma, and neurosensory hearing loss, whereas Clouston syndrome is characterized by nail dystrophy, hypotrichosis, and palmoplantar keratoderma. The present paper presents a Polish patient with sporadic KID syndrome caused by the mutation of p.Asp50Asn in GJB2. The patient encountered difficulties in obtaining a correct diagnosis. The other case presented is that of a family with Clouston syndrome (caused by p.Gly11Arg mutation in GJB6), who are the first reported patients of Polish origin suffering from this disorder. Phenotype diversity among patients with the same genotypes reported to date is also summarized. The conclusion is that proper diagnosis of these syndromes is still challenging and should always be followed by molecular verification.
连接蛋白属于间隙连接蛋白家族,该家族通过在相邻细胞中形成通道实现细胞间的直接通讯。连接蛋白基因突变会导致多种人类疾病,在少数情况下会引发皮肤疾病。两种罕见的常染色体显性综合征——角膜炎 - 鱼鳞病 - 耳聋(KID)综合征和汗孔性外胚层发育不良(克劳斯综合征)的临床表现存在显著差异,它们分别由GJB2和GJB6基因突变引起。尽管在这两种情况下,都观察到了同一家族蛋白功能异常以及一些重叠的临床特征(指甲营养不良、脱发和掌跖角化病)。KID综合征的特征是进行性血管化角膜炎、鱼鳞病样红皮角化病和神经性听力丧失,而克劳斯综合征的特征是指甲营养不良、毛发稀少和掌跖角化病。本文介绍了一名因GJB2基因p.Asp50Asn突变导致散发性KID综合征的波兰患者。该患者在获得正确诊断方面遇到困难。另一例是一个患有克劳斯综合征(由GJB6基因p.Gly11Arg突变引起)的家族,他们是首例有报道的波兰籍该疾病患者。本文还总结了迄今为止报道的相同基因型患者之间的表型多样性。结论是,对这些综合征进行正确诊断仍然具有挑战性,并且始终应随后进行分子验证。