中国多发性骨软骨瘤和强直性脊柱炎家族中IL17RC错义变异的鉴定

IF 2.5 3区 生物学 Q2 GENETICS & HEREDITY
Yingchun Zheng, Xuewu Wei, Zhongzhi Gan, Mingming Zhang, Zongrui Shen, Shunfei Ma, Yihao Huang, Fei He, Jian Wang, Fu Xiong
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引用次数: 0

摘要

强直性脊柱炎(AS)是一种慢性进行性炎性关节炎,涉及免疫系统和骨骼系统的疾病。多发性骨软骨瘤(MO)是一种罕见的骨骼疾病,临床表现多样,以多发良性外生瘤为特征。在此,我们研究了一个中国hla - b27阴性AS合并MO的家庭。采用全外显子组测序(WES)和Sanger测序筛选和鉴定致病基因。进行了体外功能分析,并分析了发病相关的白细胞介素(IL)-17受体C (IL17RC)突变,以研究其对表型的影响。利用WES鉴定了致病基因EXT1中已知的错义突变NM_000127.3: C .1019 G > a (p.a g340his),该突变可导致MO。此外,IL17RC基因中的错义突变NM_153461.3: C .1067 C > T(p.s r356met)被鉴定为该家族中as或脊椎关节炎症状的潜在原因。在体外过表达突变体IL17RC会降低其表达,升高IL17RA的表达,这与这两个基因在患者体内的表达一致。在机制上,突变体IL17RC增强了NF-κB通路的激活。这项研究增加了我们对这些疾病的发病机制和进展的理解。我们的发现拓宽了与AS中NF-κB通路相关的非hla - b基因的危险因素。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Identification of an IL17RC missense variant in a Chinese family with multiple osteochondromas and ankylosing spondylitis.

Ankylosing spondylitis (AS) is a chronic and progressive inflammatory arthritis involving disorders of both the immune and skeletal systems. Multiple osteochondromas (MO) is a rare skeletal disorder with a variety of clinical manifestations characterized by multiple benign exostoses. Here, we investigate a Chinese family with HLA-B27-negative AS complicated with MO. Whole-exome sequencing (WES) and Sanger sequencing were used to screen and identify the pathogenic gene. In vitro functional analysis was performed, and a pathogenesis-associated interleukin (IL)-17 receptor C (IL17RC) mutation was analyzed to investigate its effect on phenotypes. WES was used to identify a known missense mutation, NM_000127.3:c.1019 G > A(p.Arg340His), in the pathogenic gene EXT1 that is causal for MO. Moreover, a missense mutation, NM_153461.3:c.1067 C > T(p.Thr356Met), in the IL17RC gene was identified as potentially responsible for AS or spondyloarthritis symptoms in this family. In vitro over-expression of mutant IL17RC decreased its expression and increased the expression of IL17RA, consistent with the expression of these two genes in patients. Mechanistically, mutant IL17RC enhanced the activation of the NF-κB pathway. This study increases our understanding of the pathogenesis and progression of these diseases. Our findings broaden the risk factors in non-HLA-B genes associated with the NF-κB pathway in AS.

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来源期刊
Journal of Human Genetics
Journal of Human Genetics 生物-遗传学
CiteScore
7.20
自引率
0.00%
发文量
101
审稿时长
4-8 weeks
期刊介绍: The Journal of Human Genetics is an international journal publishing articles on human genetics, including medical genetics and human genome analysis. It covers all aspects of human genetics, including molecular genetics, clinical genetics, behavioral genetics, immunogenetics, pharmacogenomics, population genetics, functional genomics, epigenetics, genetic counseling and gene therapy. Articles on the following areas are especially welcome: genetic factors of monogenic and complex disorders, genome-wide association studies, genetic epidemiology, cancer genetics, personal genomics, genotype-phenotype relationships and genome diversity.
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