Mitchell Zoe H, den Hoed Joery, Claassen Willemijn, Demurtas Martina, Deelen Laura, Campeau Philippe M, Liu Karen, Fisher Simon E, Trizzino Marco
Department of Life Sciences, Imperial College London, London, UK.
Language and Genetics Department, Max Planck Institute for Psycholinguistic, Nijmegen, The Netherlands.
EMBO Rep. 2025 Aug 20. doi: 10.1038/s44319-025-00555-w.
Pathogenic genetic variants in the NuRD component CHD3 cause Snijders Blok-Campeau Syndrome, a neurodevelopmental disorder manifesting with intellectual disability and craniofacial anomalies. To investigate the role of CHD3 in craniofacial development, we differentiated control and CHD3-depleted human-induced pluripotent stem cells into cranial neural crest cells (CNCCs). In control lines, CHD3 is upregulated in early stages of CNCC specification, where it enhances the BMP signalling response by opening chromatin at BMP-responsive cis-regulatory elements and by increasing expression of BMP-responsive transcription factors, including DLX paralogs. CHD3 loss leads to repression of BMP target genes and loss of chromatin accessibility at cis-regulatory elements usually bound by BMP-responsive factors, causing an imbalance between BMP and Wnt signalling. Consequently, the CNCC specification fails, replaced by aberrant early-mesoderm identity, which can be partially rescued by titrating Wnt levels. Our findings highlight a novel role for CHD3 as a pivotal regulator of BMP signalling, essential for proper neural crest specification and craniofacial development. Moreover, these results suggest a molecular mechanism for the craniofacial anomalies of Snijders Blok-Campeau Syndrome.
核小体重塑去乙酰化酶(NuRD)复合物成分CHD3中的致病性基因变异会导致斯奈德·布洛克 - 坎波综合征,这是一种神经发育障碍,表现为智力残疾和颅面畸形。为了研究CHD3在颅面发育中的作用,我们将对照和CHD3缺失的人诱导多能干细胞分化为颅神经嵴细胞(CNCCs)。在对照细胞系中,CHD3在CNCC特化的早期阶段上调,通过在BMP反应性顺式调控元件处打开染色质以及增加包括DLX旁系同源物在内的BMP反应性转录因子的表达来增强BMP信号反应。CHD3缺失导致BMP靶基因的抑制以及通常由BMP反应因子结合的顺式调控元件处染色质可及性的丧失,从而导致BMP和Wnt信号之间的失衡。因此,CNCC特化失败,被异常的早期中胚层身份所取代,通过调节Wnt水平可部分挽救这种情况。我们的研究结果突出了CHD3作为BMP信号关键调节因子的新作用,这对于正确的神经嵴特化和颅面发育至关重要。此外,这些结果提示了斯奈德·布洛克 - 坎波综合征颅面畸形的分子机制。