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角膜炎-鱼鳞病-耳聋综合征:携带p.Asp50Asn GJB2突变患者的表型异质性及治疗前景

Keratitis-ichthyosis-deafness syndrome: Phenotypic heterogeneity and treatment perspective of patients with p.Asp50Asn GJB2 mutation.

作者信息

Asgari Tina, Naji Mahtab, Mansouri Parvin, Mahmoudi Hamidreza, Zabihi Masoud, Youssefian Leila, Mahdavi Mohammadreza, Naraghi Zahra Safaei, Zeinali Sirous, Vahidnezhad Hassan, Uitto Jouni

机构信息

Department of Biology, Science, and Research Branch, Islamic Azad University, Tehran, Iran.

Department of Dermatology and Cutaneous Biology, Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.

出版信息

Dermatol Ther. 2020 Nov;33(6):e14493. doi: 10.1111/dth.14493. Epub 2020 Nov 23.

Abstract

Keratitis-ichthyosis-deafness (KID) syndrome is caused by mutations in the GJB2 gene encoding connexin 26, a component of transmembrane hemichannels which form gap junction channels, critical for cell-cell communication. Here, we report two patients from two distinct families with KID syndrome with the same GJB2 mutation (p.Asp50Asn); in both cases the mutation was de novo, as the parents depicted the wild-type allele only. The patients' cutaneous manifestations were strikingly different illustrating the wide spectrum of phenotype of these patients, even with the same GJB2 mutation. One of the patients was treated with acitretin with dramatic improvement in his skin findings, illustrating the role of oral acitretin in treatment of patients with KID syndrome. Collectively, these patients attest to the phenotypic spectrum of KID syndrome, with therapeutic perspective.

摘要

角膜炎-鱼鳞病-耳聋(KID)综合征是由编码连接蛋白26的GJB2基因突变引起的,连接蛋白26是跨膜半通道的一个组成部分,跨膜半通道形成对细胞间通讯至关重要的间隙连接通道。在此,我们报告了来自两个不同家庭的两名患有KID综合征且具有相同GJB2突变(p.Asp50Asn)的患者;在这两个病例中,该突变均为新发突变,因为父母仅表现出野生型等位基因。患者的皮肤表现显著不同,这说明了即使具有相同的GJB2突变,这些患者的表型范围也很广。其中一名患者接受了阿维A治疗,其皮肤症状有显著改善,这说明了口服阿维A在治疗KID综合征患者中的作用。总体而言,这些患者证实了KID综合征的表型范围以及治疗前景。

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