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FOXP2 基因内小缺失与儿童言语失用症和构音障碍有关。

Small intragenic deletion in FOXP2 associated with childhood apraxia of speech and dysarthria.

机构信息

Department of Paediatrics, The Royal Children's Hospital, The University of Melbourne, Parkville, Victoria, Australia.

出版信息

Am J Med Genet A. 2013 Sep;161A(9):2321-6. doi: 10.1002/ajmg.a.36055. Epub 2013 Aug 5.

Abstract

Relatively little is known about the neurobiological basis of speech disorders although genetic determinants are increasingly recognized. The first gene for primary speech disorder was FOXP2, identified in a large, informative family with verbal and oral dyspraxia. Subsequently, many de novo and familial cases with a severe speech disorder associated with FOXP2 mutations have been reported. These mutations include sequencing alterations, translocations, uniparental disomy, and genomic copy number variants. We studied eight probands with speech disorder and their families. Family members were phenotyped using a comprehensive assessment of speech, oral motor function, language, literacy skills, and cognition. Coding regions of FOXP2 were screened to identify novel variants. Segregation of the variant was determined in the probands' families. Variants were identified in two probands. One child with severe motor speech disorder had a small de novo intragenic FOXP2 deletion. His phenotype included features of childhood apraxia of speech and dysarthria, oral motor dyspraxia, receptive and expressive language disorder, and literacy difficulties. The other variant was found in a family in two of three family members with stuttering, and also in the mother with oral motor impairment. This variant was considered a benign polymorphism as it was predicted to be non-pathogenic with in silico tools and found in database controls. This is the first report of a small intragenic deletion of FOXP2 that is likely to be the cause of severe motor speech disorder associated with language and literacy problems.

摘要

虽然越来越多的人认识到遗传因素在言语障碍中起着重要作用,但人们对言语障碍的神经生物学基础仍知之甚少。第一个主要言语障碍的致病基因是 FOXP2,该基因在一个有言语和口部运动障碍的大型信息家族中被发现。随后,许多具有严重言语障碍并与 FOXP2 突变相关的新发和家族性病例被报道。这些突变包括测序改变、易位、单亲二体、基因组拷贝数变异。我们研究了 8 名言语障碍患者及其家属。使用言语、口腔运动功能、语言、读写能力和认知的全面评估来对家庭成员进行表型分析。FOXP2 的编码区被筛选以鉴定新的变异。在先证者的家族中确定变异的分离情况。在两名先证者中发现了变异。一名患有严重运动性言语障碍的儿童存在 FOXP2 基因内小的新生缺失。他的表型包括儿童言语失用症和构音障碍、口腔运动性运动障碍、接受性和表达性语言障碍以及读写困难的特征。另一个变异在一个有口吃的三个家庭成员中的两个以及有口腔运动障碍的母亲中发现。由于该变异被预测为非致病性的,并且在计算机工具和数据库对照中发现,因此被认为是良性多态性。这是第一个报道的 FOXP2 基因内小缺失的病例,可能是与语言和读写问题相关的严重运动性言语障碍的原因。

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