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使用分子核型分析技术对孤立性和复杂性自闭症谱系障碍的诊断效能及新变异

Diagnostic efficacy and new variants in isolated and complex autism spectrum disorder using molecular karyotyping.

作者信息

Lovrečić Luca, Rajar Polona, Volk Marija, Bertok Sara, Gnidovec Stražišar Barbara, Osredkar Damjan, Jekovec Vrhovšek Maja, Peterlin Borut

机构信息

Clinical Institute of Medical Genetics, University Medical Centre Ljubljana, Slajmerjeva 4, 1000, Ljubljana, Slovenia.

Department of Pediatric Endocrinology, Diabetes and Metabolic Diseases, Division of Paediatrics, University Medical Centre Ljubljana, Bohoriceva 28, 1000, Ljubljana, Slovenia.

出版信息

J Appl Genet. 2018 May;59(2):179-185. doi: 10.1007/s13353-018-0440-y. Epub 2018 Mar 21.

Abstract

Autism spectrum disorder (ASD) is a group of the neurodevelopment disorders presenting as an isolated ASD or more complex forms, where a broader clinical phenotype comprised of developmental delay and intellectual disability is present. Both the isolated and complex forms have a significant causal genetic component and submicroscopic genomic copy number variations (CNV) are the most common identifiable genetic factor in these patients. The data on microarray testing in ASD cohorts are still accumulating and novel loci are often identified; therefore, we aimed to evaluate the diagnostic efficacy of the method and the relevance of implementing it into routine genetic testing in ASD patients. A genome-wide CNV analysis using the Agilent microarrays was performed in a group of 150 individuals with an isolated or complex ASD. Altogether, 11 (7.3%) pathogenic CNVs and 15 (10.0%) variants of unknown significance (VOUS) were identified, with the highest proportion of pathogenic CNVs in the subgroup of the complex ASD patients (14.3%). An interesting case of previously unreported partial UPF3B gene deletion was identified among the pathogenic CNVs. Among the CNVs with unknown significance, four VOUS involved genes with possible correlation to ASD, namely genes SNTG2, PARK2, CADPS2 and NLGN4X. The diagnostic efficacy of aCGH in our cohort was comparable with those of the previously reported and identified an important proportion of genetic ASD cases. Despite the continuum of published studies on the CNV testing in ASD cohorts, a considerable number of VOUS CNVs is still being identified, namely 10.0% in our study.

摘要

自闭症谱系障碍(ASD)是一组神经发育障碍,表现为孤立性ASD或更复杂的形式,伴有发育迟缓及智力残疾等更广泛的临床表型。孤立性和复杂性形式均有显著的因果遗传成分,亚微观基因组拷贝数变异(CNV)是这些患者中最常见的可识别遗传因素。ASD队列中微阵列检测的数据仍在积累,新的基因座也经常被发现;因此,我们旨在评估该方法的诊断效能以及将其应用于ASD患者常规基因检测的相关性。我们对150例患有孤立性或复杂性ASD的个体进行了使用安捷伦微阵列的全基因组CNV分析。共鉴定出11个(7.3%)致病性CNV和15个(10.0%)意义未明的变异(VOUS),其中致病性CNV在复杂性ASD患者亚组中的比例最高(14.3%)。在致病性CNV中发现了1例之前未报道过的UPF3B基因部分缺失的有趣病例。在意义未明的CNV中,有4个VOUS涉及可能与ASD相关的基因,即SNTG2、PARK2、CADPS2和NLGN4X基因。我们队列中aCGH的诊断效能与之前报道的相当,并鉴定出了相当比例的遗传性ASD病例。尽管关于ASD队列中CNV检测的已发表研究不断,但仍有相当数量的VOUS CNV被发现,在我们的研究中为10.0%。

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