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发育异常性椎弓崩裂由硫酸软骨素硫酸酯转运蛋白基因的突变引起。

Dysplastic spondylolysis is caused by mutations in the diastrophic dysplasia sulfate transporter gene.

作者信息

Cai Tao, Yang Liu, Cai Wanshi, Guo Sen, Yu Ping, Li Jinchen, Hu Xueyu, Yan Ming, Shao Qianzhi, Jin Yan, Sun Zhong Sheng, Luo Zhuo-Jing

机构信息

Institute of Genomic Medicine, Wenzhou Medical University, Wenzhou 325000, China;

Institute of Orthopaedics, Xijing Hospital, Fourth Military Medical University, Xi'an 710000, China;

出版信息

Proc Natl Acad Sci U S A. 2015 Jun 30;112(26):8064-9. doi: 10.1073/pnas.1502454112. Epub 2015 Jun 15.

Abstract

Spondylolysis is a fracture in part of the vertebra with a reported prevalence of about 3-6% in the general population. Genetic etiology of this disorder remains unknown. The present study was aimed at identifying genomic mutations in patients with dysplastic spondylolysis as well as the potential pathogenesis of the abnormalities. Whole-exome sequencing and functional analysis were performed for patients with spondylolysis. We identified a novel heterozygous mutation (c.2286A > T; p.D673V) in the sulfate transporter gene SLC26A2 in five affected subjects of a Chinese family. Two additional mutations (e.g., c.1922A > G; p.H641R and g.18654T > C in the intron 1) in the gene were identified by screening a cohort of 30 unrelated patients with the disease. In situ hybridization analysis showed that SLC26A2 is abundantly expressed in the lumbosacral spine of the mouse embryo at day 14.5. Sulfate uptake activities in CHO cells transfected with mutant SLC26A2 were dramatically reduced compared with the wild type, confirming the pathogenicity of the two missense mutations. Further analysis of the gene-disease network revealed a convergent pathogenic network for the development of lumbosacral spine. To our knowledge, our findings provide the first identification of autosomal dominant SLC26A2 mutations in patients with dysplastic spondylolysis, suggesting a new clinical entity in the pathogenesis of chondrodysplasia involving lumbosacral spine. The analysis of the gene-disease network may shed new light on the study of patients with dysplastic spondylolysis and spondylolisthesis as well as high-risk individuals who are asymptomatic.

摘要

峡部裂是椎骨部分的骨折,据报道在普通人群中的患病率约为3%-6%。这种疾病的遗传病因尚不清楚。本研究旨在确定发育异常性峡部裂患者的基因组突变以及异常的潜在发病机制。对峡部裂患者进行了全外显子测序和功能分析。我们在中国一个家族的5名受累个体中,在硫酸盐转运蛋白基因SLC26A2中发现了一种新的杂合突变(c.2286A>T;p.D673V)。通过对30名无关疾病患者的队列筛查,在该基因中又鉴定出另外两个突变(例如,c.1922A>G;p.H641R和内含子1中的g.18654T>C)。原位杂交分析表明,SLC26A2在小鼠胚胎第14.5天的腰骶部脊柱中大量表达。与野生型相比,转染突变型SLC26A2的CHO细胞中的硫酸盐摄取活性显著降低,证实了这两个错义突变的致病性。对基因-疾病网络的进一步分析揭示了腰骶部脊柱发育的一个收敛致病网络。据我们所知,我们的发现首次鉴定出发育异常性峡部裂患者中的常染色体显性SLC26A2突变,提示涉及腰骶部脊柱的软骨发育异常发病机制中的一种新的临床实体。基因-疾病网络分析可能为发育异常性峡部裂和腰椎滑脱患者以及无症状高危个体的研究提供新的线索。

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