MECP2基因R306X突变导致摩洛哥患者非典型Rett综合征:一例报告

IF 1.9 Q3 PATHOLOGY Clinical Pathology Pub Date : 2022-09-16 eCollection Date: 2022-01-01 DOI:10.1177/2632010X221124269
Wafaa Bouzroud, Amal Tazzite, Sarah Berrada, Bouchaïb Gazzaz, Hind Dehbi
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引用次数: 0

摘要

Rett综合征(RTT)是一种罕见的x连锁综合征,主要影响女孩。它的特征是一种严重的进行性神经发育障碍,伴有神经退化和自闭症谱系特征。Rett综合征具有广泛的表型谱。它的范围从由公认标准定义的经典Rett综合征到具有与其他综合征(如Angelman综合征)相似症状的非典型病例。首例摩洛哥女童携带MECP2(甲基- cpg结合蛋白2)基因R306X突变,表现为Rett综合征。她表现出自闭症消退、行为停滞、癫痫、无动机的笑声和颅面畸形。全外显子组测序显示无义突变(R306X),导致截断,无功能的MECP2蛋白。Rett综合征和Angelman综合征之间的重叠表型谱已被描述,MECP2基因与UBE3A(泛素蛋白连接酶E3A)基因途径之间可能存在相互作用,但尚未得到证实。强烈建议在非典型病例中进行广泛的遗传分析,以确保准确诊断并改善患者管理和遗传咨询。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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R306X Mutation in the MECP2 Gene Causes an Atypical Rett Syndrome in a Moroccan Patient: A Case Report.

Rett syndrome (RTT) is a rare X-linked syndrome that predominantly affects girls. It is characterized by a severe and progressive neurodevelopmental disorder with neurological regression and autism spectrum features. The Rett syndrome is associated with a broad phenotypic spectrum. It ranges from a classical Rett syndrome defined by well-established criteria to atypical cases with symptoms similar to other syndromes, such as Angelman syndrome. The first case of a Moroccan female child carrying a R306X mutation in the MECP2 (Methyl-CpG-Binding Protein 2) gene, with an unusual manifestation of Rett syndrome, is presented here. She showed autistic regression, behavioral stagnation, epilepsy, unmotivated laughter, and craniofacial dysmorphia. Whole exome sequencing revealed a nonsense mutation (R306X), resulting in a truncated, nonfunctional MECP2 protein. The overlapping phenotypic spectrums between Rett and Angelman syndromes have been described, and an interaction between the MECP2 gene and the UBE3A (Ubiquitin Protein Ligase E3A) gene pathways is possible but has not yet been proven. An extensive genetic analysis is highly recommended in atypical cases to ensure an accurate diagnosis and to improve patient management and genetic counseling.

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来源期刊
Clinical Pathology
Clinical Pathology PATHOLOGY-
CiteScore
2.20
自引率
7.70%
发文量
66
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