| Literature DB >> 25692795 |
Eissa A Faqeih1, Laila Bastaki, Rasim Ozgur Rosti, Emily G Spencer, AbdulAli P Zada, Mohammad A M Saleh, Kyongmi Um, Joseph G Gleeson.
Abstract
Microcephaly-capillary malformation syndrome (MIC-CAP syndrome) is a newly recognized autosomal recessive congenital neurocutaneous central nervous system disorder characterized by severe microcephaly, early-onset seizures, profound psychomotor disability, and multiple cutaneous capillary lesions. In addition, affected patients have variable dysmorphic facial features and hypoplastic distal phalanges. It is distinctively caused by mutations in a newly characterized gene, STAMBP, encoding the deubiquitinating (DUB) isopeptidase that has a key role in cell surface receptor-mediated endocytosis and sorting. Herein, we describe an Arab family of two siblings with classic features of MIC-CAP syndrome that harbor a novel predicted splice mutation in STAMBP, which additionally display previously unreported findings of congenital hypothyroidism and alopecia areata.Entities:
Keywords: MIC-CAP syndrome; STAMBP gene; alopecia areata; capillary malformation; congenital hypothyroidism
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Year: 2015 PMID: 25692795 DOI: 10.1002/ajmg.a.36782
Source DB: PubMed Journal: Am J Med Genet A ISSN: 1552-4825 Impact factor: 2.802