Literature DB >> 24115482

A patient with a unique frameshift mutation in GPC3, causing Simpson-Golabi-Behmel syndrome, presenting with craniosynostosis, penoscrotal hypospadias, and a large prostatic utricle.

Diana D Villarreal1, Humberto Villarreal, Ana Maria Paez, Dennis Peppas, Jane Lynch, Elizabeth Roeder, George C Powers.   

Abstract

We present a Hispanic male with the clinical and molecular diagnosis of Simpson-Golabi-Behmel syndrome (SGBS). The patient was born with multiple anomalies not entirely typical of SGBS patients, including penoscrotal hypospadias, a large prostatic utricle, and left coronal craniosynostosis. In addition, he demonstrated endocrine anomalies including a low random cortisol level suspicious for adrenal insufficiency and low testosterone level. To our knowledge, this is the first report of a prostatic utricle in SGBS and the second report of craniosynostosis. The unique disease-causing mutation likely arose de novo in the mother. It is a deletion-insertion that leads to a frameshift at the p.p. S359 [corrected] residue of GPC3 and a premature stop codon after five more amino acids. p. S359 [corrected] is the same residue that is normally cleaved by the Furin convertase, although the significance of this novel mutation with respect to the patient's multiple anomalies is unknown. We present this case as the perinatal course of a patient with unique features of SGBS and a confirmed molecular diagnosis.
© 2013 Wiley Periodicals, Inc.

Entities:  

Keywords:  Simpson-Golabi-Behmel syndrome; adrenal insufficiency; ambiguous genitalia; coronal synostosis; craniosynostosis; diaphragmatic hernia; glypican 3; hypospadias; large for gestational age; prostatic utricle

Mesh:

Substances:

Year:  2013        PMID: 24115482     DOI: 10.1002/ajmg.a.36086

Source DB:  PubMed          Journal:  Am J Med Genet A        ISSN: 1552-4825            Impact factor:   2.802


  5 in total

Review 1.  The influence of genetics in congenital diaphragmatic hernia.

Authors:  Lan Yu; Rebecca R Hernan; Julia Wynn; Wendy K Chung
Journal:  Semin Perinatol       Date:  2019-08-01       Impact factor: 3.300

2.  Closing the Gap: Genetic and Genomic Continuum from Syndromic to Nonsyndromic Craniosynostoses.

Authors:  Yann Heuzé; Gregory Holmes; Inga Peter; Joan T Richtsmeier; Ethylin Wang Jabs
Journal:  Curr Genet Med Rep       Date:  2014-09-01

Review 3.  Simpson-Golabi-Behmel syndrome types I and II.

Authors:  Jair Tenorio; Pedro Arias; Víctor Martínez-Glez; Fernando Santos; Sixto García-Miñaur; Julián Nevado; Pablo Lapunzina
Journal:  Orphanet J Rare Dis       Date:  2014-09-20       Impact factor: 4.123

4.  Novel GPC3 Gene Mutation in Simpson-Golabi-Behmel Syndrome with Endocrine Anomalies: A Case Report.

Authors:  W Bu; M Zhu; S Li; H Liu; X Liu
Journal:  Balkan J Med Genet       Date:  2022-06-05       Impact factor: 0.810

5.  Simpson-Golabi-Behmel syndrome type 1 with subclinical hypothyroidism: A case report.

Authors:  Jing Zhang; Kai Mu; Haiyan Xu; Yuehua Guo; Zhijie Liu; Liling Wang; Jiahui Li; Fengjuan Zhang; Yan Kou; Xin Yuan
Journal:  Medicine (Baltimore)       Date:  2019-10       Impact factor: 1.817

  5 in total

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