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MED13 基因突变会表现出歌舞伎综合征样特征吗?

Could the MED13 mutations manifest as a Kabuki-like syndrome?

机构信息

University of Trieste, Trieste, Italy.

Institute for Maternal and Child Health IRCCS "Burlo Garofolo", Trieste, Italy.

出版信息

Am J Med Genet A. 2021 Feb;185(2):584-590. doi: 10.1002/ajmg.a.61994. Epub 2020 Nov 30.

Abstract

MED13-related disorder is a new neurodevelopmental disorder recently described in literature, which belongs to the group of CDK8-kinase module genes-associated conditions. It is characterized by variable intellectual disability and/or developmental delays, especially in language. Autism spectrum disorder (ASD), attention deficit hyperactivity disorder (ADHD), eye or vision problems, hypotonia, mild congenital hearth abnormalities and dysmorphisms have been described among individuals with MED13 mutations. We report the case of a 13-year-old girl who received a previous clinical diagnosis of Kabuki syndrome (KS) without mutations in classic KS genes. After a whole exome sequencing (WES) analysis a de novo missense mutation in MED13 (c.C979T; p.Pro327Ser) was found. This variant has been once described in literature as accountable for a novel neurodevelopmental disorder. The aim of this report is to improve clinical delineation of MED13-related condition and to explore differences and similarities between KS spectrum and MED13-related disorders.

摘要

MED13 相关障碍是一种新的神经发育障碍,最近在文献中有所描述,属于 CDK8-激酶模块基因相关疾病。其特征为智力障碍和/或发育迟缓,尤其是语言方面。已有研究报道携带 MED13 突变的个体存在自闭症谱系障碍(ASD)、注意缺陷多动障碍(ADHD)、眼部或视力问题、肌张力低下、轻度先天性心脏异常和畸形等情况。我们报告了一例 13 岁女孩的病例,其先前临床诊断为歌舞伎综合征(KS),但经典 KS 基因无突变。经过全外显子组测序(WES)分析,发现 MED13 中存在一个新生错义突变(c.C979T;p.Pro327Ser)。该变异曾在文献中被描述为可导致一种新的神经发育障碍。本报告旨在提高对 MED13 相关疾病的临床描述,并探讨 KS 综合征谱和 MED13 相关疾病之间的差异和相似之处。

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