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无汗性外胚叶发育不良,不伴无汗性外胚叶发育不良。

Focal dermal hypoplasia without focal dermal hypoplasia.

机构信息

Departamento de Genética, Centro de Investigaciones Regionales Dr. Hideyo Noguchi, Mérida, Yucatán, México.

出版信息

Am J Med Genet A. 2014 Mar;164A(3):778-81. doi: 10.1002/ajmg.a.36341. Epub 2013 Dec 19.

Abstract

Focal dermal hypoplasia (FDH; Goltz-Gorlin syndrome) is an X-linked dominant disorder affecting mainly tissues of ectodermal and mesodermal origin. The phenotype is characterized by hypoplastic linear skin lesions, eye malformations, hair and teeth anomalies, and multiple limbs malformations. The disorder is caused by PORCN mutations. Here we describe a mother and daughter with FDH in whom a c.938T>G in PORCN was detected. Neither of the two had FDH, but otherwise the phenotype was classical. Focal skin hypoplasia is a hallmark of FDH but the present family indicates that FDH should also be considered in absence of this skin manifestation.

摘要

局限性皮肤发育不良(FDH;Goltz-Gorlin 综合征)是一种 X 连锁显性遗传疾病,主要影响外胚层和中胚层来源的组织。其表型特征为发育不良的线性皮肤损伤、眼部畸形、毛发和牙齿异常以及四肢多发性畸形。该疾病由 PORCN 突变引起。本文描述了一例 FDH 母女病例,在其 PORCN 基因中检测到 c.938T>G 突变。母女俩均无 FDH 皮肤表现,但其他表型均符合经典 FDH 特征。局限性皮肤发育不良是 FDH 的一个标志性特征,但本家族病例提示,在无皮肤表现的情况下也应考虑 FDH 的可能。

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