Suppr超能文献

一个X连锁显性点状软骨发育不良家族中的外显率降低。

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

作者信息

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

机构信息

Institut für Humangenetik, Universität zu Lübeck, Ratzeburger Allee 160, 23538 Lübeck, Germany.

出版信息

Eur J Med Genet. 2007 Sep-Oct;50(5):392-8. doi: 10.1016/j.ejmg.2007.05.004. Epub 2007 Jun 3.

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.

摘要

X连锁显性点状软骨发育不良(康拉迪-许纳曼病,CDPX2)的特征为身材矮小、骨骺点状钙化、白内障、鱼鳞病样红皮病和头皮斑片状脱发。该疾病由编码3β-羟基类固醇-δ(8),δ(7)-异构酶的依莫帕米结合蛋白(EBP)基因突变引起。疾病严重程度的家族内差异是CDPX2的一个已知特征,可能由X染色体失活偏倚所致。我们报告了一名患有CDPX2典型症状的女性胎儿,如四肢短小、轴后多指畸形、鱼鳞病样皮肤损害和点状钙化。EBP基因的分子遗传学分析显示一个无义突变(c.328C>T,p.R110X),该突变先前在一名CDPX2患者和另一名仅身体一侧受累且被误诊为CHILD综合征的女性患者中检测到。令人惊讶的是,我们胎儿的母亲携带相同突变,但没有任何CDPX2的体征。X染色体失活研究未发现母亲或胎儿存在失活偏倚的任何证据。

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验