Department of Pediatrics, Columbia University Irving Medical Center, New York, NY 10032, USA; Department of Systems Biology, Columbia University Irving Medical Center, New York, NY 10032, USA.
Department of Pediatrics, Columbia University Irving Medical Center, New York, NY 10032, USA.
Am J Hum Genet. 2024 Nov 7;111(11):2362-2381. doi: 10.1016/j.ajhg.2024.08.024. Epub 2024 Sep 26.
Congenital diaphragmatic hernia (CDH) is a severe congenital anomaly often accompanied by other structural anomalies and/or neurobehavioral manifestations. Rare de novo protein-coding variants and copy-number variations contribute to CDH in the population. However, most individuals with CDH remain genetically undiagnosed. Here, we perform integrated de novo and common-variant analyses using 1,469 CDH individuals, including 1,064 child-parent trios and 6,133 ancestry-matched, unaffected controls for the genome-wide association study. We identify candidate CDH variants in 15 genes, including eight novel genes, through deleterious de novo variants. We further identify two genomic loci contributing to CDH risk through common variants with similar effect sizes among Europeans and Latinx. Both loci are in putative transcriptional regulatory regions of developmental patterning genes. Estimated heritability in common variants is ∼19%. Strikingly, there is no significant difference in estimated polygenic risk scores between isolated and complex CDH or between individuals harboring deleterious de novo variants and individuals without these variants. The data support a polygenic model as part of the CDH genetic architecture.
先天性膈疝 (CDH) 是一种严重的先天性异常,常伴有其他结构异常和/或神经行为表现。罕见的新生蛋白编码变异和拷贝数变异导致人群中出现 CDH。然而,大多数 CDH 患者的遗传仍未得到诊断。在这里,我们使用包括 1064 个儿童-父母三核苷酸和 6133 个亲缘匹配的无影响对照在内的 1469 名 CDH 个体,进行了全基因组关联研究的新生成和常见变异综合分析。我们通过有害的新生变异,在 15 个基因中鉴定出候选 CDH 变异,包括 8 个新基因。我们进一步通过欧洲人和拉丁裔中具有相似效应大小的常见变异,鉴定出两个与 CDH 风险相关的基因组位点。这两个位点都位于发育模式基因的假定转录调控区域。常见变异的估计遗传率约为 19%。引人注目的是,孤立性和复杂性 CDH 之间,或携带有害新生变异的个体与没有这些变异的个体之间,估计的多基因风险评分没有显著差异。这些数据支持多基因模型作为 CDH 遗传结构的一部分。