Department of Pediatric Surgery, Guangzhou Institute of Pediatrics, Guangzhou Women and Children's Medical Center, Guangzhou Medical University, Guangzhou, Guangdong, China.
J Cell Mol Med. 2018 Apr;22(4):2190-2199. doi: 10.1111/jcmm.13498. Epub 2018 Jan 29.
Hirschsprung disease (HSCR) is a genetic disorder characterized by the absence of enteric ganglia. There are more than 15 genes identified as contributed to HSCR by family-based or population-based approaches. However, these findings were not fulfilled to explain the heritability of most sporadic cases. In this study, using 1470 HSCR and 1473 control subjects in South Chinese population, we replicated two variants in NRG1 (rs16879552, P = 1.05E-04 and rs7835688, P = 1.19E-07), and further clarified the two replicated SNPs were more essential for patients with short-segment aganglionosis (SHSCR) (P = 2.37E-05). We also tried to replicate the most prominent signal (rs7785360) in AUTS2, which was a potential susceptibility gene with HSCR. In our results, in terms of individual association, marginal effect was observed to affect the HSCR patients following recessive model (P = 0.089). Noteworthy, significant intergenic synergistic effect between rs16879552 (NRG1) and rs7785360 (AUTS2) was identified through cross-validation by logistic regression (P = 2.45E-03, OR = 1.53) and multifactor dimensionality reduction (MDR, P < 0.0001, OR = 1.77). Significant correlation was observed between expression of these two genes in the normal segments of the colons (P = 0.018), together with differential expression of these genes between aganglionic colonic segments and normal colonic segments of the HSCR patients (P value for AUTS2 <0.0001, P value for NRG1 = 0.0243). Although functional evaluation is required, we supply new evidence for the NRG1 to HSCR and raised up a new susceptibility gene AUTS2 to a specific symptom for the disease.
先天性巨结肠症(HSCR)是一种以肠神经节缺失为特征的遗传疾病。通过基于家系或基于人群的方法,已经确定了超过 15 个基因与 HSCR 有关。然而,这些发现并不能解释大多数散发性病例的遗传性。在这项研究中,我们使用了华南地区的 1470 名 HSCR 患者和 1473 名对照受试者,复制了 NRG1 中的两个变体(rs16879552,P=1.05E-04 和 rs7835688,P=1.19E-07),并进一步阐明这两个复制的 SNP 对短节段无神经节细胞症(SHSCR)患者更为重要(P=2.37E-05)。我们还试图复制 AUTS2 中最显著的信号(rs7785360),该基因是 HSCR 的一个潜在易感基因。在我们的结果中,就个体关联而言,在隐性模型下,观察到边缘效应影响 HSCR 患者(P=0.089)。值得注意的是,通过逻辑回归交叉验证(P=2.45E-03,OR=1.53)和多因子降维分析(MDR,P<0.0001,OR=1.77),鉴定到了 rs16879552(NRG1)和 rs7785360(AUTS2)之间显著的基因间协同作用。在 HSCR 患者的正常结肠段中,观察到这两个基因的表达之间存在显著相关性(P=0.018),并且在无神经节细胞的结肠段和 HSCR 患者的正常结肠段之间,这两个基因的表达存在差异(AUTS2 的 P 值<0.0001,NRG1 的 P 值=0.0243)。尽管需要进行功能评估,但我们为 NRG1 与 HSCR 之间的关系提供了新的证据,并提出了一个新的易感基因 AUTS2,用于解释该疾病的特定症状。