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自显性营养不良性大疱性表皮松解症:东南亚 5 例患者的临床、分子和遗传学特征的首次报告。

Self-improving dystrophic epidermolysis bullosa: First report of clinical, molecular, and genetic characterization of five patients from Southeast Asia.

机构信息

Dermatology Service, KK Women's & Children's Hospital, Singapore, Singapore.

Skin Research Institute of Singapore, Agency for Science, Technology and Research (A*STAR), Singapore, Singapore.

出版信息

Am J Med Genet A. 2021 Feb;185(2):625-630. doi: 10.1002/ajmg.a.61975. Epub 2020 Nov 30.

Abstract

Self-improving dystrophic epidermolysis bullosa is a rare subtype of dystrophic epidermolysis bullosa (DEB) characterized by significant improvement in skin fragility within the first few years of life. Genetic inheritance has previously been reported as autosomal dominant or recessive with both forms harboring mutations in COL7A1. To date, there have been no reports of this rare clinical entity from various Southeast Asian ethnicities. Here, we describe the clinical and molecular features of five patients from the Southeast Asia region who presented with predominantly acral-distributed blisters and erosions in the first few days of life. Blistering resolved over several months, without appearance of new blisters. By immunofluorescence, intraepidermal retention of Type VII collagen was observed in all patient skin biopsies when investigated with antibody staining. Genetic analysis of four patients revealed pathogenic variants in COL7A1 which have not been previously reported. The clinical diagnosis in these rare patients is confirmed with molecular histology and genetic characterization.

摘要

自限性营养不良型大疱性表皮松解症是一种罕见的营养不良型大疱性表皮松解症(DEB)亚型,其特征是在生命的头几年皮肤脆弱性显著改善。遗传方式以前被报道为常染色体显性或隐性遗传,两种形式都在 COL7A1 中存在突变。迄今为止,尚未有来自不同东南亚种族的这种罕见临床实体的报告。在这里,我们描述了来自东南亚地区的五名患者的临床和分子特征,他们在生命的头几天主要表现为肢端分布的水疱和糜烂。水疱在数月内消退,没有出现新的水疱。通过免疫荧光染色,所有患者的皮肤活检均观察到 VII 型胶原在表皮内的保留。对四名患者的基因分析显示 COL7A1 存在先前未报道的致病性变异。这些罕见患者的临床诊断通过分子组织学和基因特征得到证实。

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