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  • 标题:A non-mosaic PORCN mutation in a male with severe congenital anomalies overlapping focal dermal hypoplasia
  • 本地全文:下载
  • 作者:Simran Madan ; Wei Liu ; James T. Lu
  • 期刊名称:Molecular Genetics and Metabolism Reports
  • 印刷版ISSN:2214-4269
  • 出版年度:2017
  • 卷号:12
  • 页码:57-61
  • DOI:10.1016/j.ymgmr.2017.06.002
  • 出版社:Elsevier B.V.
  • 摘要:

    Mutations in the PORCN gene cause the X-linked dominant condition focal dermal hypoplasia (FDH). Features of FDH include striated pigmentation of the skin, ocular and skeletal malformations. FDH is generally associated with in utero lethality in non-mosaic males and most of the currently reported male patients show mosaicism due to de novo post-zygotic mutations in the PORCN gene. There is only one previous report of a surviving male with an inherited mutation in the PORCN gene. Here, we report two male siblings with multiple malformations including skeletal, ocular and renal defects overlapping with FDH. A novel PORCN mutation (p.Ser250Phe) was identified in a non-mosaic, hemizygous state in one of the siblings who survived to 8 years of age. The mother is a heterozygous carrier, has a random X-inactivation pattern and is asymptomatic. Findings unusual for FDH include dysplastic clavicles and bilateral Tessier IV facial clefts. This is the second case report of a non-mosaic PORCN mutation in a male individual with multiple congenital anomalies. While the pathogenicity of this mutation remains to be further investigated, the survival of a male with a non-mosaic mutation in PORCN is suggestive of a functionally mild mutation leading to an X-linked recessive mode of inheritance.

  • 关键词:Focal dermal hypoplasia ; Goltz syndrome ; PORCN ; Non-mosaic ; Micro-opthalmia
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