Institute of Cytology and Genetics SB RAS, Novosibirsk, Russia.
Novosibirsk State University, Novosibirsk, Russia.
Sci Rep. 2024 Mar 4;14(1):5288. doi: 10.1038/s41598-024-52545-y.
Structural variations are a pervasive feature of human genomes, and there is growing recognition of their role in disease development through their impact on spatial chromatin architecture. This understanding has led us to investigate the clinical significance of CNVs in noncoding regions that influence TAD structures. In this study, we focused on the Epb41l4a locus, which contains a highly conserved TAD boundary present in both human chromosome 5 and mouse chromosome 18, and its association with neurodevelopmental phenotypes. Analysis of human data from the DECIPHER database indicates that CNVs within this locus, including both deletions and duplications, are often observed alongside neurological abnormalities, such as dyslexia and intellectual disability, although there is not enough evidence of a direct correlation or causative relationship. To investigate these possible associations, we generated mouse models with deletion and inversion mutations at this locus and carried out RNA-seq analysis to elucidate gene expression changes. We found that modifications in the Epb41l4a TAD boundary led to dysregulation of the Nrep gene, which plays a crucial role in nervous system development. These findings underscore the potential pathogenicity of these CNVs and highlight the crucial role of spatial genome architecture in gene expression regulation.
结构变异是人类基因组的普遍特征,越来越多的人认识到它们通过影响空间染色质结构在疾病发展中的作用。这一认识促使我们研究影响 TAD 结构的非编码区 CNVs 的临床意义。在这项研究中,我们专注于 Epb41l4a 基因座,该基因座包含一个高度保守的 TAD 边界,存在于人类 5 号染色体和小鼠 18 号染色体中,及其与神经发育表型的关联。对 DECIPHER 数据库中人类数据的分析表明,该基因座内的 CNVs,包括缺失和重复,常与神经发育异常(如阅读障碍和智力障碍)同时发生,尽管没有足够的证据表明存在直接相关性或因果关系。为了研究这些可能的关联,我们在该基因座上生成了缺失和倒位突变的小鼠模型,并进行了 RNA-seq 分析以阐明基因表达变化。我们发现,Epb41l4a TAD 边界的改变导致了 Nrep 基因的失调,Nrep 基因在神经系统发育中起着关键作用。这些发现强调了这些 CNVs 的潜在致病性,并突出了空间基因组结构在基因表达调控中的关键作用。