Université Paris Descartes, INSERM U, Hôpital Necker-Enfants Malades, France.
Hum Mutat. 2011 Jan;32(1):70-2. doi: 10.1002/humu.21384.
Hypohidrotic and anhidrotic ectodermal dysplasia (HED/EDA) is a rare genodermatosis characterized by abnormal development of sweat glands, teeth, and hair. Three disease-causing genes have been hitherto identified, namely, (1) EDA1 accounting for X-linked forms, (2) EDAR, and (3) EDARADD, causing both autosomal dominant and recessive forms. Recently, WNT10A gene was identified as responsible for various autosomal recessive forms of ectodermal dysplasias, including onycho-odonto-dermal dysplasia (OODD) and Schöpf-Schulz-Passarge syndrome. We systematically studied EDA1, EDAR, EDARADD, and WNT10A genes in a large cohort of 65 unrelated patients, of which 61 presented with HED/EDA. A total of 50 mutations (including 32 novel mutations) accounted for 60/65 cases in our series. These four genes accounted for 92% (56/61 patients) of HED/EDA cases: (1) the EDA1 gene was the most common disease-causing gene (58% of cases), (2)WNT10A and EDAR were each responsible for 16% of cases. Moreover, a novel disease locus for dominant HED/EDA mapped to chromosome 14q12-q13.1. Although no clinical differences between patients carrying EDA1, EDAR, or EDARADD mutations could be identified, patients harboring WNT10A mutations displayed distinctive clinical features (marked dental phenotype, no facial dysmorphism), helping to decide which gene should be first investigated in HED/EDA.
汗少型和无汗型外胚层发育不良(HED/EDA)是一种罕见的遗传性皮肤病,其特征为汗腺、牙齿和毛发发育异常。迄今为止,已经确定了三个致病基因,即(1)EDA1,负责 X 连锁形式,(2)EDAR,和(3)EDARADD,导致常染色体显性和隐性形式。最近,WNT10A 基因被确定为各种常染色体隐性外胚层发育不良的原因,包括甲-牙-齿-毛发发育不良(OODD)和 Schöpf-Schulz-Passarge 综合征。我们系统地研究了 EDA1、EDAR、EDARADD 和 WNT10A 基因在一个由 65 名无血缘关系的患者组成的大队列中,其中 61 名患者表现为 HED/EDA。共有 50 个突变(包括 32 个新突变)占我们系列中的 60/65 例。这四个基因占 HED/EDA 病例的 92%(56/61 例):(1)EDA1 基因是最常见的致病基因(58%的病例),(2)WNT10A 和 EDAR 各占 16%的病例。此外,一个新的显性 HED/EDA 疾病基因座定位于 14q12-q13.1。虽然携带 EDA1、EDAR 或 EDARADD 突变的患者之间没有明显的临床差异,但携带 WNT10A 突变的患者表现出独特的临床特征(明显的牙齿表型,无面部畸形),有助于决定在 HED/EDA 中首先检查哪个基因。
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