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中国一个家族中TNNI2基因的一种新型错义突变导致1型远端关节挛缩症。

A novel missense mutation of TNNI2 in a Chinese family cause distal arthrogryposis type 1.

作者信息

Wang Bo, Zheng Zhaojing, Wang Zhigang, Zhang Xiaoqing, Yang Haiou, Cai Haiqing, Fu Qihua

机构信息

Department of Laboratory Medicine, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, P.R. China.

Department of Pediatric Orthopedic, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, Shanghai, P.R. China.

出版信息

Am J Med Genet A. 2016 Jan;170A(1):135-41. doi: 10.1002/ajmg.a.37391. Epub 2015 Sep 16.

Abstract

The distal arthrogryposis (DA) syndromes are a group of disorders characterized by congenital contractures of limbs. According to phenotypical characteristics, DA syndromes have been clinically classified into 10 types. Currently, at least nine disease causing genes have been identified for different types of DA. Here, we report a 3-generation Chinese pedigree with three DA affected members. We performed whole exome sequencing on two affected and one unaffected individuals of this family and successfully identified a novel missense mutation in TNNI2 as the pathogenic mutation. The TNNI2 gene encodes a subunit of the troponin complex, a contractile machinery of the muscle. The mutation p.F178C that could change the H-bond formation of a neighboring residue occurs at a highly conserved position, suggesting that this variation probably affects the TNNI2 protein function. Our study also demonstrates the power of whole exome sequencing in causal mutation identification for phenotypically variable and genetically heterogeneous disorders.

摘要

远端关节挛缩症(DA)综合征是一组以肢体先天性挛缩为特征的疾病。根据表型特征,DA综合征在临床上已被分为10种类型。目前,已针对不同类型的DA鉴定出至少9个致病基因。在此,我们报告一个三代中国家系,其中有三名成员患有DA。我们对该家系中两名患者和一名未患病个体进行了全外显子组测序,并成功鉴定出TNNI2基因中的一个新错义突变作为致病突变。TNNI2基因编码肌钙蛋白复合体的一个亚基,肌钙蛋白复合体是肌肉的收缩机制。发生在高度保守位置的p.F178C突变可能会改变相邻残基的氢键形成,这表明这种变异可能影响TNNI2蛋白的功能。我们的研究还证明了全外显子组测序在鉴定表型可变和基因异质性疾病的致病突变方面的作用。

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