Literature DB >> 17625999

Reduced penetrance in a family with X-linked dominant chondrodysplasia punctata.

Yorck Hellenbroich1, Karl-Heinz Grzeschik, Martin Krapp, Tiantom Jarutat, Christa Lehrmann-Petersen, Karin Buiting, Gabriele Gillessen-Kaesbach.   

Abstract

X-linked dominant chondrodysplasia punctata (Conradi-Hünermann disease, CDPX2) is characterised by short stature, stippled epiphyses, cataracts, ichthyosiform erythroderma and patchy alopecia of the scalp. The disorder is caused by mutations within the emopamil binding protein (EBP) gene encoding a 3beta-hydroxysteroid-Delta(8),Delta(7)-isomerase. The intrafamilial variation of disease severity is a known feature of CDPX2 probably caused by skewed X-inactivation. We report on a female fetus with typical symptoms of CDPX2 such as short limbs, postaxial polydactyly, ichthyotic skin lesions and punctate calcifications. Molecular genetic analysis of the EBP gene revealed a nonsense mutation (c.328C>T, p.R110X), which was previously detected in one CDPX2 patient and in a second female patient, who was only affected on one body side and erroneously diagnosed as CHILD syndrome. Surprisingly, the mother of our fetus carries the same mutation without having any signs of CDPX2. X-inactivation studies did not reveal any evidence of skewing neither in the mother nor in the fetus.

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Year:  2007        PMID: 17625999     DOI: 10.1016/j.ejmg.2007.05.004

Source DB:  PubMed          Journal:  Eur J Med Genet        ISSN: 1769-7212            Impact factor:   2.708


  2 in total

Review 1.  Radiographic features of the skeleton in disorders of post-squalene cholesterol biosynthesis.

Authors:  Massimiliano Rossi; Christine M Hall; Raymonde Bouvier; Sophie Collardeau-Frachon; Frédérique Le Breton; Martine Bucourt; Marie Pierre Cordier; Christine Vianey-Saban; Giancarlo Parenti; Generoso Andria; Martine Le Merrer; Patrick Edery; Amaka C Offiah
Journal:  Pediatr Radiol       Date:  2015-02-03

2.  New splicing pathogenic variant in EBP causing extreme familial variability of Conradi-Hünermann-Happle Syndrome.

Authors:  Mathilde Pacault; Marie Vincent; Thomas Besnard; Caroline Kannengiesser; Claire Bénéteau; Sébastien Barbarot; Xénia Latypova; Khaldia Belabbas; Antonin Lamazière; Norbert Winer; Madeleine Joubert; Stéphane Bézieau; Bertrand Isidor; Sandra Mercier; Mathilde Nizon; Stéphanie Leclerc-Mercier; Smail Hadj-Rabia; Fabienne Dufernez
Journal:  Eur J Hum Genet       Date:  2018-08-22       Impact factor: 4.246

  2 in total

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