KRT10的L12连接子结构域变异是导致非典型表皮溶解性鱼鳞病的原因。

IF 2.9 3区 医学 Q2 DERMATOLOGY Journal of Dermatology Pub Date : 2024-07-29 DOI:10.1111/1346-8138.17395
J. J. A. J. van der Velden, M. W. van Gisbergen, M. A. F. Kamps, R. Janssen, G. F. H. Diercks, P. M. Steijlen, M. van Geel, M. C. Bolling
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引用次数: 0

摘要

表皮溶解性鱼鳞病(EI)是一种先天性鱼鳞病,其特征是出生时出现红斑和水疱,随后角化过度。EI是由分别编码角蛋白1(KRT1)和角蛋白10(KRT10)的基因KRT1和KRT10的致病变体引起的,主要通过常染色体显性遗传进行传播,但也有由KRT10的无义变体引起的隐性遗传。角蛋白形成中间丝网络,是细胞骨架的结构成分,赋予皮肤强度和韧性。我们报告了三例因 KRT10 L12 连接子结构域的致病变异而导致的轻度 EI 病例。据我们所知,这是首次在 KRT10 中发现 L12 连接子结构域致病变异导致 EI。本研究的目的是确定 EI 患者的 KRT1 或 KRT10 基因变异。为了确定在 KRT10 中发现的变异的致病性,我们对所有患者和可用的家庭成员进行了临床评估。基因分析采用桑格测序法进行。将含有野生型或突变型 KRT10 的载体转染到 HaCaT 细胞中,并用高分辨率共聚焦显微镜进行分析。KRT10 的遗传分析在家族 1 中发现了一个 c.910G>A p.(Val304Met) 的新发杂合变体,在家族 2 中发现了一个 c.911T>C p.(Val304Ala) 的家族杂合变体,在家族 3 中发现了一个 c.917T>C p.(Met306Thr) 的家族杂合变体。与野生型序列相比,KRT10 中的错义变异体在体外形成聚集体的研究仅表明,KRT10 网络中的聚集体形成非常轻微,且无法量化。我们报告了 KRT10 L12 连接子结构域中的三种不同的新型错义变体,这些变体出现在一种类似脱皮综合征的非典型、较轻型 EI 患者身上。
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Variants in the L12 linker domain of KRT10 are causal to atypical epidermolytic ichthyosis

Epidermolytic ichthyosis (EI) is a type of congenital ichthyosis, characterized by erythema and blistering at birth followed by hyperkeratosis. EI is caused by pathogenic variants in the genes KRT1 and KRT10, encoding the proteins keratin 1 (KRT1) and keratin 10 (KRT10), respectively, and is primarily transmitted by autosomal-dominant inheritance, although recessive inheritance caused by nonsense variants in KRT10 is also described. The keratins form a network of intermediate filaments and are a structural component of the cytoskeleton, giving strength and resilience to the skin. We present three cases of mild EI caused by pathogenic KRT10 variations in the L12 linker domain. To our knowledge, this is the first time L12 linker domain pathogenic variants are identified in KRT10 for EI. The aim of this study was to identify gene variants for patients with EI in KRT1 or KRT10. To establish the pathogenicity of the found variations in KRT10, we evaluated all patients and available family members clinically. Genetic analyses were performed using Sanger sequencing. Vectors containing wild-type or mutated forms of KRT10 were transfected into HaCaT cells and analyzed by high-resolution confocal microscopy. Genetic analysis of KRT10 identified a heterozygous de novo variant c.910G>A p.(Val304Met) in family 1, a familial heterozygous variant c.911T>C p.(Val304Ala) in family 2, and a familial heterozygous variant c.917T>C p.(Met306Thr) in family 3. All identified missense variants were located in the L12 linker domain of KRT10. In vitro study of aggregate formation of the missense variants in KRT10 only showed a very mild and not quantifiable aggregate formation in the KRT10 network, compared with the wild-type sequence. We report three different novel missense variants in the L12 linker domain of KRT10 in patients with an atypical, milder form of EI resembling peeling skin syndrome.

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来源期刊
Journal of Dermatology
Journal of Dermatology 医学-皮肤病学
CiteScore
4.60
自引率
9.70%
发文量
368
审稿时长
4-8 weeks
期刊介绍: The Journal of Dermatology is the official peer-reviewed publication of the Japanese Dermatological Association and the Asian Dermatological Association. The journal aims to provide a forum for the exchange of information about new and significant research in dermatology and to promote the discipline of dermatology in Japan and throughout the world. Research articles are supplemented by reviews, theoretical articles, special features, commentaries, book reviews and proceedings of workshops and conferences. Preliminary or short reports and letters to the editor of two printed pages or less will be published as soon as possible. Papers in all fields of dermatology will be considered.
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