Research Institute of Molecular Pathology, Vienna BioCenter, 1030 Vienna, Austria.
Vienna BioCenter PhD Program, Doctoral School of the University of Vienna and Medical University of Vienna, A-1030 Vienna, Austria.
Proc Natl Acad Sci U S A. 2022 May 3;119(18):e2201029119. doi: 10.1073/pnas.2201029119. Epub 2022 Apr 27.
Cornelia de Lange syndrome (CdLS) is a developmental multisystem disorder frequently associated with mutations in NIPBL. CdLS is thought to arise from developmental gene regulation defects, but how NIPBL mutations cause these is unknown. Here we show that several NIPBL mutations impair the DNA loop extrusion activity of cohesin. Because this activity is required for the formation of chromatin loops and topologically associating domains, which have important roles in gene regulation, our results suggest that defects in cohesin-mediated loop extrusion contribute to the etiology of CdLS by altering interactions between developmental genes and their enhancers.
Cornelia de Lange 综合征(CdLS)是一种常见的多系统发育障碍疾病,通常与 NIPBL 基因突变有关。CdLS 被认为是由于发育基因调控缺陷引起的,但 NIPBL 突变如何导致这些缺陷尚不清楚。在这里,我们表明几种 NIPBL 突变会损害黏连蛋白的 DNA 环外推活性。由于这种活性对于染色质环和拓扑关联域的形成是必需的,而这些结构在基因调控中具有重要作用,因此我们的结果表明,黏连蛋白介导的环外推缺陷通过改变发育基因与其增强子之间的相互作用,导致 CdLS 的发生。