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一组伊朗患者中II型瓦登伯格综合征的全面基因与临床评估

A Comprehensive Genetic and Clinical Evaluation of Waardenburg Syndrome Type II in a Set of Iranian Patients.

作者信息

Jalilian Nazanin, Tabatabaiefar Mohammad Amin, Yazdanpanah Mahboubeh, Darabi Elham, Bahrami Tayyeb, Zekri Ali, Noori-Daloii Mohammad Reza

机构信息

Department of Clinical biochemistry, School of Medicine, Kermanshah University of Medical Sciences, Kermanshah, Iran.

Department of Genetics and Molecular Biology, School of Medicine, Isfahan University of Medical Sciences, Isfahan, Iran.

出版信息

Int J Mol Cell Med. 2018 Winter;7(1):17-23. doi: 10.22088/IJMCM.BUMS.7.1.17. Epub 2018 Mar 27.

Abstract

Waardenburg syndrome (WS) is a neurocristopathy with an autosomal dominant mode of inheritance, and considerable clinical and genetic heterogeneity. WS type II is the most common type of WS in many populations presenting with sensorineural hearing impairment, heterochromia iridis, hypoplastic blue eye, and pigmentary abnormalities of the hair and skin. To date, mutations of , , and have been implicated in the pathogenesis of WS2. Although different pathogenic mutations have been reported in many ethnic groups, the data on Iranian WS2 patients is insufficient. 31 WS2 patients, including 22 men and 9 women from 14 families were included. Waardenburg consortium guidelines were employed for WS2 diagnosis. WS2 patients underwent screening for , , and mutations using direct sequencing and MLPA analysis. Clinical evaluation revealed prominent phenotypic variability in Iranian WS2 patients. Sensorineural hearing impairment and heterochromia iridis were the most common features (67% and 45%, respectively), whereas anosmia was the least frequent phenotype. Molecular analysis revealed a heterozygous c.640C>T (p.R214X) in and a heterozygous gross deletion in the study population. Our data help illuminate the phenotypic and genotypic spectrum of WS2 in an Iranian series of patients, and could have implications for the genetic counseling of WS in Iran.

摘要

瓦登伯革氏综合征(WS)是一种具有常染色体显性遗传模式的神经嵴病,存在显著的临床和遗传异质性。在许多人群中,II型瓦登伯革氏综合征(WS2)是WS最常见的类型,表现为感音神经性听力障碍、虹膜异色症、蓝色眼睛发育不全以及毛发和皮肤色素异常。迄今为止, 、 和 的突变与WS2的发病机制有关。尽管在许多种族群体中都报道了不同的致病突变,但关于伊朗WS2患者的数据并不充分。本研究纳入了来自14个家庭的31例WS2患者,其中包括22名男性和9名女性。采用瓦登伯革氏综合征协作组指南对WS2进行诊断。使用直接测序和多重连接探针扩增(MLPA)分析对WS2患者进行 、 和 突变筛查。临床评估显示伊朗WS2患者存在显著的表型变异性。感音神经性听力障碍和虹膜异色症是最常见的特征(分别为67%和45%),而嗅觉丧失是最不常见的表型。分子分析在研究人群中发现了一个 杂合的c.640C>T(p.R214X)突变和一个 杂合的大片段缺失。我们的数据有助于阐明伊朗系列患者中WS2的表型和基因型谱,并可能对伊朗WS的遗传咨询产生影响。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7c55/6134422/946c51fd8daa/ijmcm-7-017-g001.jpg

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