Department of Molecular and Cellular Biochemistry.
Department of Oral and Maxillofacial Oncology and Surgery, and.
JCI Insight. 2024 Jun 10;9(11):e175486. doi: 10.1172/jci.insight.175486.
The transcription factor SRY-related HMG box 9 (Sox9) is essential for chondrogenesis. Mutations in and around SOX9 cause campomelic dysplasia (CD) characterized by skeletal malformations. Although the function of Sox9 in this context is well studied, the mechanisms that regulate Sox9 expression in chondrocytes remain to be elucidated. Here, we have used genome-wide profiling to identify 2 Sox9 enhancers located in a proximal breakpoint cluster responsible for CD. Enhancer activity of E308 (located 308 kb 5' upstream) and E160 (located 160 kb 5' upstream) correlated with Sox9 expression levels, and both enhancers showed a synergistic effect in vitro. While single deletions in mice had no apparent effect, simultaneous deletion of both E308 and E160 caused a dwarf phenotype, concomitant with a reduction of Sox9 expression in chondrocytes. Moreover, bone morphogenetic protein 2-dependent chondrocyte differentiation of limb bud mesenchymal cells was severely attenuated in E308/E160 deletion mice. Finally, we found that an open chromatin region upstream of the Sox9 gene was reorganized in the E308/E160 deletion mice to partially compensate for the loss of E308 and E160. In conclusion, our findings reveal a mechanism of Sox9 gene regulation in chondrocytes that might aid in our understanding of the pathophysiology of skeletal disorders.
转录因子 SRY 相关高迁移率族盒 9(Sox9)对于软骨生成至关重要。SOX9 中的突变和其周围的突变导致卡梅隆发育不良(CD),其特征是骨骼畸形。尽管 Sox9 在这种情况下的功能已得到充分研究,但调节软骨细胞中 Sox9 表达的机制仍有待阐明。在这里,我们使用全基因组分析鉴定了两个 Sox9 增强子,它们位于负责 CD 的近端断点簇中。E308(位于 5'上游 308kb 处)和 E160(位于 5'上游 160kb 处)的增强子活性与 Sox9 表达水平相关,并且两者在体外均表现出协同作用。虽然小鼠中的单个缺失没有明显的影响,但同时缺失 E308 和 E160 会导致矮小型表型,同时伴随着软骨细胞中 Sox9 表达的减少。此外,E308/E160 缺失小鼠的肢体芽间充质细胞中骨形态发生蛋白 2 依赖性软骨细胞分化受到严重抑制。最后,我们发现 Sox9 基因上游的一个开放染色质区域在 E308/E160 缺失小鼠中被重新组织,以部分补偿 E308 和 E160 的缺失。总之,我们的发现揭示了软骨细胞中 Sox9 基因调控的一种机制,这可能有助于我们理解骨骼疾病的病理生理学。