【由UBE2A基因大片段缺失引起的Nascimento型综合征性x连锁智力发育障碍大家族1例报告及文献复习】。

Q3 Medicine
Dan Xu, Jia-Yang Xie, Xiao-Li Zhang, Meng-Yue Wang, Man-Man Chu, Rui Han, Jun-Ling Wang, Xiao-Li Li, Tian-Ming Jia
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引用次数: 0

摘要

本文报道了一个Nascimento型综合征型x连锁智力发育障碍(MRXSN)大家族的临床特征和基因突变类型,涉及3代9人,并进行文献复习。该家族共有9人出现智力低下、异常相等类似表现,其中4人已去世。基因检测显示先证者缺失UBE2A基因外显子2-3,该基因遗传自母亲。荧光定量聚合酶链反应显示先证者及其叔父UBE2A基因外显子2-3缺失;先证者的母亲、祖母和曾祖母存在UBE2A基因外显子2-3的杂合缺失;先证者的父亲、姐姐和姨妈的UBE2A基因外显子2-3拷贝数正常。文献报道的34例患者临床表型多样,UBE2A基因突变(22/ 34.65%)和大片段缺失(12/ 34.35%)是主要的突变类型。中重度智力障碍(34/ 34,100 %)、言语语言障碍(33/ 34,97 %)和异常相(32/ 34,94 %)是MRXSN患者的主要临床表现。该病具有明显的表型异质性,早期诊断有助于优化产前和产后管理,以改善生殖结果。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
[A large family of Nascimento form of syndromic X-linked intellectual developmental disorder caused by large segment deletion of the UBE2A gene: a case report and literature review].

This article reports the clinical features and gene mutation types of a large family with Nascimento form of syndromic X-linked intellectual developmental disorder (MRXSN), involving 9 individuals across 3 generations, and a literature review was conducted. In this family, 9 individuals had similar manifestations including mental retardation and unusual facies, and 4 of them had passed away. Genetic testing showed that the proband had the deletion of exons 2-3 of the UBE2A gene, which was inherited from the mother. Fluorescent quantitative polymerase chain reaction showed that the proband and his uncle had the deletion of exons 2-3 of the UBE2A gene; the proband's mother, grandmother, and great-aunt had a heterozygous deletion of exons 2-3 of the UBE2A gene; the proband's father, sister, and aunt had a normal copy number of exons 2-3 of the UBE2A gene. The 34 patients reported in the literature had diverse clinical phenotypes, and UBE2A gene mutations (22/34, 65%) and large fragment deletions (12/34, 35%) were the main mutation types. Moderate to severe mental retardation (34/34, 100%), speech and language impairment (33/34, 97%), and unusual facies (32/34, 94%) were the main clinical manifestations of MRXSN patients. The disease has obvious phenotypic heterogeneity, and early diagnosis facilitates optimal prenatal and postnatal management to improve reproductive outcomes.

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来源期刊
中国当代儿科杂志
中国当代儿科杂志 Medicine-Pediatrics, Perinatology and Child Health
CiteScore
1.50
自引率
0.00%
发文量
5006
期刊介绍: The Chinese Journal of Contemporary Pediatrics (CJCP) is a peer-reviewed open access periodical in the field of pediatrics that is sponsored by the Central South University/Xiangya Hospital of Central South University and under the auspices of the Ministry of Education of China. It is cited as a source in the scientific and technological papers of Chinese journals, the Chinese Science Citation Database (CSCD), and is one of the core Chinese periodicals in the Peking University Library. CJCP has been indexed by MEDLINE/PubMed/PMC of the American National Library, American Chemical Abstracts (CA), Holland Medical Abstracts (EM), Western Pacific Region Index Medicus (WPRIM), Scopus and EBSCO. It is a monthly periodical published on the 15th of every month, and is distributed both at home and overseas. The Chinese series publication number is CN 43-1301/R;ISSN 1008-8830. The tenet of CJCP is to “reflect the latest advances and be open to the world”. The periodical reports the most recent advances in the contemporary pediatric field. The majority of the readership is pediatric doctors and researchers.
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