病例报告:阿尔法6整合素紊乱症在儿童期出现,伴有指甲发育不良和甲沟炎,但无脆性或牛皮癣病史。

IF 1.2 Q4 GENETICS & HEREDITY
Global Medical Genetics Pub Date : 2024-10-16 eCollection Date: 2024-12-01 DOI:10.1055/s-0044-1791804
Alayna N Zalesny, Sarah Gunter, Charles A Williams
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引用次数: 0

摘要

我们报告了一名患有幽门闭锁的 7 岁女孩,但她没有先天性表皮松解症或皮肤脆性。她在 8 个月大时出现指甲发育不良,整个童年时期都患有甲沟炎和轻度指甲肥大症。全外显子组测序显示,她的α6整合素(ITGA6)发生了双侧突变:p. Q139*和R153W。ITGA6 通常与 beta4 整合素(ITGB4)形成蛋白异源二聚体,该二聚体参与将基底皮肤细胞固定在细胞外基质上。众所周知,每个基因的双倍突变都会导致大疱性表皮松解症和幽门闭锁。然而,该患儿的皮肤表面正常,没有任何皮肤脆弱的迹象。在文献检索中,我们发现了 11 个涉及 ITGA6 基因突变的病例,所有病例都有皮肤表皮松解病变。因此,本病例是第一个在幽门闭锁和指甲发育不良的情况下显示没有表皮松解症表型的病例,从而丰富了 ITGA6 疾病的表型。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Case Report: Alpha6 Integrin Disorder Presenting in Childhood with Nail Dysplasia and Onycholysis But No History of Fragile or Bullous Skin Changes.

We report a 7-year-old girl born with pyloric atresia but without congenital epidermolysis bullosa or skin fragility. Nail dysplasia developed at age 8 months and throughout childhood she suffered from onycholysis and mild nail hypertrophy. Whole-exome sequencing demonstrated biallelic mutations in alpha6 integrin (ITGA6): p. Q139* and R153W. ITGA6 normally forms a protein heterodimer with beta4 integrin (ITGB4), and this dimer participates in anchoring the basal skin cells to the extracellular matrix. Biallelic mutations in each gene are well known to cause epidermolysis bullosa and pyloric atresia. However, this child had ostensibly normal skin without any evidence of skin fragility. In a literature search, we identified 11 cases involving ITGA6 mutations, and all had epidermolysis skin changes. Thus, this case adds to the reported phenotype of ITGA6 disease since it is the first to show absence of an epidermolysis bullosa phenotype in the setting of pyloric atresia and nail dysplasia.

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来源期刊
Global Medical Genetics
Global Medical Genetics GENETICS & HEREDITY-
自引率
11.80%
发文量
30
审稿时长
14 weeks
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