Asim Ambreen, Agarwal Sarita, Panigrahi Inusha, Sarangi Aditya Narayan, Muthuswamy Srinivasan, Kapoor Aditya
Department of Genetics, Sanjay Gandhi Postgraduate Institute of Medical Sciences (SGPGIMS), Lucknow 226014, India.
Department of Genetics, Sanjay Gandhi Postgraduate Institute of Medical Sciences (SGPGIMS), Lucknow 226014, India.
Gene. 2018 Jan 30;641:180-185. doi: 10.1016/j.gene.2017.10.044. Epub 2017 Oct 18.
Congenital heart defects (CHD) are seen in around 40% of the Down syndrome patients. Atrioventricular Septal Defect (AVSD) or endocardial cushion defect is commonest form of CHD in these children. CRELD1 gene is implicated in causation of sporadic AVSD. In the present study, we evaluated the association and significance of CRELD1 variants with AVSD in Down syndrome (DS) patients. Sequencing was done in blood samples from 3 groups: group I (DS with AVSD), group II (DS without AVSD) and group III (non-syndromic AVSD cases). Twenty two variants in CRELD1 gene were identified, comprising of sixteen novel and six previously reported variants. However, on the basis of sequence, as well as structure analysis, the variant c.973G>A(p.Glu325Lys) variant was identified only in DS having AVSD group which was predicted to have significant effects on calcium binding of putative CRELD1 protein. Since CRELD1 gene acts as a regulator of calcineurin/NFATc1 signaling which is crucial for the regulation of cardiac development by dephosphorylation of the transcription factor, NFAT(nuclear factor of activated T cells),in cytoplasm, the variation in cb-EGF-like calcium binding domain in CRELD1 protein is likely to have pathogenic consequences. Thus, we conclude that the CRELD1 gene is likely to have a major role in causation of AVSD phenotype in selected DS patients.
约40%的唐氏综合征患者患有先天性心脏病(CHD)。房室间隔缺损(AVSD)或心内膜垫缺损是这些儿童中最常见的CHD形式。CRELD1基因与散发性AVSD的病因有关。在本研究中,我们评估了CRELD1基因变异与唐氏综合征(DS)患者AVSD的关联及意义。对三组血液样本进行了测序:第一组(患有AVSD的DS患者)、第二组(未患AVSD的DS患者)和第三组(非综合征性AVSD病例)。在CRELD1基因中鉴定出22种变异,包括16种新变异和6种先前报道的变异。然而,基于序列以及结构分析,仅在患有AVSD的DS组中鉴定出变异c.973G>A(p.Glu325Lys),预计该变异对假定的CRELD1蛋白的钙结合有显著影响。由于CRELD1基因作为钙调神经磷酸酶/NFATc1信号的调节因子,通过使转录因子NFAT(活化T细胞核因子)在细胞质中去磷酸化,对心脏发育的调节至关重要,因此CRELD1蛋白中cb-EGF样钙结合结构域的变异可能具有致病后果。因此,我们得出结论,CRELD1基因可能在部分DS患者AVSD表型的病因中起主要作用。