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骨形态发生蛋白2诱导激酶基因的复发性突变与髋关节发育不良相关。

A recurrent mutation in bone morphogenetic proteins-2-inducible kinase gene is associated with developmental dysplasia of the hip.

作者信息

Zhao Lihua, Zhou Zaiwei, Wang Sun, Jiao Qing, Wu Jing, Ma Feng, Fan Lingyan, Chen Mengjie, Ying Hao

机构信息

Department of Orthopedics, Shanghai Children's Hospital, Shanghai Jiao Tong University, Shanghai 200062, P.R. China.

Laboratory of Translational Research, Institute of Pediatric Translational Medicine, Shanghai Children's Medical Center, School of Medicine, Shanghai Jiao Tong University, Shanghai 200071, P.R. China.

出版信息

Exp Ther Med. 2017 May;13(5):1773-1778. doi: 10.3892/etm.2017.4191. Epub 2017 Mar 8.

Abstract

Developmental dysplasia of the hip (DDH) is a complex disorder of the hip joint affecting 1-5‰ of newborns. While genetic influence on DDH has been long known, DDH has not been ascribed to any specific genetic event. The present study reported on variants contributing to DDH susceptibility in a family with four individuals affected across three generations. Whole-exome sequencing was performed in three affected and two unaffected individuals of a pedigree with DDH. Candidate variants were confirmed by Sanger sequencing and then validated in available family members and 37 sporadic DDH patients. Two novel heterozygous, inframe mutations causing multi-nucleotide substitution polymorphisms (c.1432_1440delCAGCAGCAG corresponding with p.Gln478_480del and c.1440_1441insCAG corresponding with p.Gln480ins) in exon 11 of chromosome 4 in bone morphogenetic proteins-2-inducible kinase (BMP2K) were identified; these were found in members of the pedigree affected by DDH and in the unaffected grandmother of the proband, who was deemed to be the carrier of potential mutations, but not in the unaffected normal control saunt of the proband. These two variants shared the same genomic coordinate but with different types of mutation in BMP2K. BMP2K is known to be associated with bone and cartridge development and heterozygous mutations were found to be present in 4/4 (100%) of the affected family members, 4/15 (26.7%) of the unaffected family members and 0/7 (0%) of the unaffected unrelated family members. Genotyping of 37 unrelated, sporadic DDH patients showed that three cases were positive for the BMP2K c.1432_1440delCAGCAGCAG variants (8.12%). These findings provided strong evidence for the role of BMP2K variants in causing DDH and demonstrated that the combination of pedigree information and next-generation sequencing is an effective method for identifying pathogenic sites associated with DDH.

摘要

发育性髋关节发育不良(DDH)是一种影响1‰至5‰新生儿的复杂髋关节疾病。虽然长期以来已知遗传因素对DDH有影响,但DDH尚未归因于任何特定的遗传事件。本研究报告了一个三代中有四名个体受影响的家族中导致DDH易感性的变异。对一个患有DDH的家系中的三名患者和两名未受影响的个体进行了全外显子组测序。候选变异通过桑格测序得到确认,然后在现有的家庭成员和37例散发性DDH患者中进行验证。在骨形态发生蛋白-2诱导激酶(BMP2K)的4号染色体外显子11中,鉴定出两个新的杂合框内突变,这些突变导致多核苷酸替代多态性(c.1432_1440delCAGCAGCAG对应于p.Gln478_480del,c.1440_1441insCAG对应于p.Gln480ins);这些突变在受DDH影响的家系成员以及先证者未受影响的祖母(被认为是潜在突变的携带者)中被发现,但在先证者未受影响的正常对照中未发现。这两个变异在BMP2K中具有相同的基因组坐标,但突变类型不同。已知BMP2K与骨骼和软骨发育有关,在4/4(100%)的受影响家庭成员、4/15(26.7%)的未受影响家庭成员和0/7(0%)的未受影响无关家庭成员中发现了杂合突变。对37例不相关的散发性DDH患者进行基因分型显示,3例患者的BMP2K c.1432_1440delCAGCAGCAG变异呈阳性(8.12%)。这些发现为BMP2K变异在导致DDH中的作用提供了有力证据,并证明家系信息与下一代测序相结合是识别与DDH相关的致病位点的有效方法。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/be7c/5443164/4cd1b5b0c26e/etm-13-05-1773-g00.jpg

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