Suppr超能文献

一个女孩患有智力障碍,无明显畸形特征,存在 MECP2 从头重复。

De novo duplication of MECP2 in a girl with mental retardation and no obvious dysmorphic features.

机构信息

Service of Genetic Medicine, University Hospitals of Geneva, Geneva, Switzerland.

出版信息

Clin Genet. 2010 Aug;78(2):175-80. doi: 10.1111/j.1399-0004.2010.01371.x. Epub 2010 Jan 5.

Abstract

Loss-of-function mutations of MECP2 are responsible for Rett syndrome (RTT), an X-linked neurodevelopmental disorder affecting mainly girls. The availability of MECP2 testing has led to the identification of such mutations in girls with atypical RTT features and the recognition of milder forms. Furthermore, duplication of the entire gene has recently been described in boys with mental retardation and recurrent infections. We describe a girl with a heterozygous de novo MECP2 duplication. The patient, at the age of 19, has mental retardation with no autistic features. She is friendly but gets frequently anxious. She has neither dysmorphic features nor malformations. Her motor development was delayed with walking at 20 months. Speech is fluid with good pronunciation but is simple and repetitive. Diagnosis was made after single-strand conformation analysis (SSCA) and multiplex ligation-dependent probe amplification (MLPA) analysis of MECP2. Array comparative genomic hybridization (aCGH) analysis showed a duplication of 29 kb including MECP2 and part of IRAK1. Fluorescent in situ hybridization (FISH) has revealed that the duplicated region is inserted near the telomere of the short arm of chromosome 10. X-chromosome inactivation in leukocyte DNA was not skewed. We conclude that it is likely that this MECP2 duplication is responsible for the mental retardation in this patient. This case broadens the phenotypic spectrum of MECP2 abnormalities with consequent implication in diagnosis and genetic counselling of girls with non-syndromic mental retardation.

摘要

MECP2 基因的功能丧失突变是导致雷特综合征(RTT)的原因,该病是一种主要影响女孩的 X 连锁神经发育障碍。由于 MECP2 检测的可用性,已经在具有非典型 RTT 特征的女孩中发现了这种突变,并认识到了更轻微的形式。此外,整个基因的重复最近也在具有智力障碍和反复感染的男孩中被描述。我们描述了一名患有杂合性新生 MECP2 重复的女孩。患者 19 岁,智力障碍但无自闭症特征。她友善但经常焦虑。她既没有畸形特征也没有畸形。她的运动发育延迟,20 个月才会走路。言语流利,发音良好,但简单且重复。在对 MECP2 进行单链构象分析(SSCA)和多重连接依赖性探针扩增(MLPA)分析后做出了诊断。比较基因组杂交分析(aCGH)显示,包括 MECP2 和 IRAK1 部分在内的 29kb 区域发生了重复。荧光原位杂交(FISH)显示,重复区域插入 10 号染色体短臂的端粒附近。白细胞 DNA 的 X 染色体失活没有偏斜。我们得出结论,很可能是这个 MECP2 重复导致了患者的智力障碍。这种情况拓宽了 MECP2 异常的表型谱,从而对非综合征性智力障碍女孩的诊断和遗传咨询产生了影响。

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