Pediatric Surgical Research Laboratories, Massachusetts General Hospital, Boston, Massachusetts 02114, USA.
Am J Med Genet A. 2012 Dec;158A(12):3148-58. doi: 10.1002/ajmg.a.35665. Epub 2012 Nov 19.
Chromosome 8p23.1 is a common hotspot associated with major congenital malformations, including congenital diaphragmatic hernia (CDH) and cardiac defects. We present findings from high-resolution arrays in patients who carry a loss (n = 18) or a gain (n = 1) of sub-band 8p23.1. We confirm a region involved in both diaphragmatic and heart malformations. Results from a novel CNVConnect algorithm, prioritizing protein-protein interactions between products of genes in the 8p23.1 hotspot and products of previously known CDH causing genes, implicated GATA4, NEIL2, and SOX7 in diaphragmatic defects. Sequence analysis of these genes in 226 chromosomally normal CDH patients, as well as in a small number of deletion 8p23.1 patients, showed rare unreported variants in the coding region; these may be contributing to the diaphragmatic phenotype. We also demonstrated that two of these three genes were expressed in the E11.5-12.5 primordial mouse diaphragm, the developmental stage at which CDH is thought to occur. This combination of bioinformatics and expression studies can be applied to other chromosomal hotspots, as well as private microdeletions or microduplications, to identify causative genes and their interaction networks.
8p23.1 号染色体是与重大先天性畸形相关的常见热点区域,包括先天性膈疝 (CDH) 和心脏缺陷。我们报告了携带 8p23.1 亚带缺失 (n=18) 或增益 (n=1) 的患者的高分辨率图谱的结果。我们证实了一个涉及膈肌和心脏畸形的区域。一种新的 CNVConnect 算法的结果,优先考虑 8p23.1 热点区域基因产物与先前已知的导致 CDH 的基因产物之间的蛋白质-蛋白质相互作用,将 GATA4、NEIL2 和 SOX7 牵连到膈肌缺陷中。在 226 例染色体正常的 CDH 患者和少数缺失 8p23.1 患者中对这些基因进行的序列分析显示,编码区存在罕见的未报道变异;这些可能导致膈肌表型。我们还表明,这三个基因中的两个在 E11.5-12.5 原始小鼠膈肌中表达,这是 CDH 发生的发育阶段。这种生物信息学和表达研究的组合可应用于其他染色体热点区域以及私人微缺失或微重复,以鉴定致病基因及其相互作用网络。