Hashmi Jamil Amjad, Almatrafi Ahmad, Latif Muhammad, Nasir Abdul, Basit Sulman
Center for Genetics and Inherited Diseases, Taibah University, Almadinah Almunawwarah, Saudi Arabia.
College of Science, Taibah University, Almadinah Almunawwarah, Saudi Arabia.
Eur J Med Genet. 2019 Feb;62(2):124-128. doi: 10.1016/j.ejmg.2018.06.013. Epub 2018 Jun 28.
We recruited a family with an affected child exhibiting features of cleidocranial dysplasia with some phenotypic variations from reported cases. Whole exome sequencing data analysis identified an 18-bps heterozygous in-frame deletion variant (c.243-260delGGCGGCTGCGGCGGCGGC) in the RUNX2 gene. Sanger sequencing validated the presence of deletion in affected individual. Initially, we considered this variant as a causal mutation for the patient's phenotype based on previous report(s). However, further analysis of variant revealed that it is present in high frequency in variety of genome variation databases. Moreover, segregation analysis discovered the presence of variant in mother as well. Furthermore, screening of population matched control individuals revealed that the variant is present in apparently healthy individuals as well. Three-dimensional structures of the wild-type and mutant RUNX2 protein (p.Ala82_Ala87del) were analysed and it was found that both wild type and mutant protein show similar secondary structure pattern. Presence of RUNX2 deletion variant (c.243-260delGGCGGCTGCGGCGGCGGC) in control individuals, its high population frequency, benign effect on the overall protein structure lead to the argument that this variant is a population polymorphism and not a pathogenic mutation.
我们招募了一个家庭,该家庭中有一个患病儿童,其表现出锁骨颅骨发育不全的特征,与已报道的病例相比存在一些表型差异。全外显子组测序数据分析在RUNX2基因中鉴定出一个18个碱基对的杂合框内缺失变异(c.243-260delGGCGGCTGCGGCGGCGGC)。桑格测序验证了患病个体中该缺失的存在。最初,基于先前的报告,我们认为这个变异是患者表型的致病突变。然而,对该变异的进一步分析表明,它在各种基因组变异数据库中以高频率存在。此外,分离分析发现母亲中也存在该变异。此外,对匹配的群体对照个体进行筛查发现,该变异在明显健康的个体中也存在。对野生型和突变型RUNX2蛋白(p.Ala82_Ala87del)的三维结构进行了分析,发现野生型和突变型蛋白都显示出相似的二级结构模式。对照个体中存在RUNX2缺失变异(c.243-260delGGCGGCTGCGGCGGCGGC),其在群体中的高频率以及对整体蛋白质结构的良性影响,使得有观点认为这个变异是一种群体多态性,而非致病突变。