MacDermot Kay D, Bonora Elena, Sykes Nuala, Coupe Anne-Marie, Lai Cecilia S L, Vernes Sonja C, Vargha-Khadem Faraneh, McKenzie Fiona, Smith Robert L, Monaco Anthony P, Fisher Simon E
Wellcome Trust Centre for Human Genetics, University of Oxford, Oxford, United Kingdom.
Am J Hum Genet. 2005 Jun;76(6):1074-80. doi: 10.1086/430841. Epub 2005 Apr 22.
FOXP2, the first gene to have been implicated in a developmental communication disorder, offers a unique entry point into neuromolecular mechanisms influencing human speech and language acquisition. In multiple members of the well-studied KE family, a heterozygous missense mutation in FOXP2 causes problems in sequencing muscle movements required for articulating speech (developmental verbal dyspraxia), accompanied by wider deficits in linguistic and grammatical processing. Chromosomal rearrangements involving this locus have also been identified. Analyses of FOXP2 coding sequence in typical forms of specific language impairment (SLI), autism, and dyslexia have not uncovered any etiological variants. However, no previous study has performed mutation screening of children with a primary diagnosis of verbal dyspraxia, the most overt feature of the disorder in affected members of the KE family. Here, we report investigations of the entire coding region of FOXP2, including alternatively spliced exons, in 49 probands affected with verbal dyspraxia. We detected variants that alter FOXP2 protein sequence in three probands. One such variant is a heterozygous nonsense mutation that yields a dramatically truncated protein product and cosegregates with speech and language difficulties in the proband, his affected sibling, and their mother. Our discovery of the first nonsense mutation in FOXP2 now opens the door for detailed investigations of neurodevelopment in people carrying different etiological variants of the gene. This endeavor will be crucial for gaining insight into the role of FOXP2 in human cognition.
FOXP2是首个被认为与发育性交流障碍有关的基因,它为研究影响人类言语和语言习得的神经分子机制提供了一个独特的切入点。在深入研究的KE家族的多名成员中,FOXP2基因的杂合错义突变导致了清晰发音所需的肌肉运动排序出现问题(发育性言语失用症),同时伴有语言和语法处理方面更广泛的缺陷。还发现了涉及该基因座的染色体重排。对特定语言障碍(SLI)、自闭症和诵读困难等典型形式中FOXP2编码序列的分析,尚未发现任何病因变体。然而,此前尚无研究对初步诊断为言语失用症的儿童进行突变筛查,而言语失用症是KE家族受影响成员中该疾病最明显的特征。在此,我们报告了对49名患有言语失用症的先证者的FOXP2整个编码区(包括可变剪接外显子)的研究。我们在三名先证者中检测到了改变FOXP2蛋白序列的变体。其中一个这样的变体是杂合无义突变,它产生了一个大幅截短的蛋白质产物,并在先证者、其患病的兄弟姐妹及其母亲中与言语和语言困难共分离。我们在FOXP2中首次发现无义突变,这为详细研究携带该基因不同病因变体的人群的神经发育打开了大门。这一努力对于深入了解FOXP2在人类认知中的作用至关重要。