a State Key Laboratory of Genetic Engineering, Department of Anthropology and Human Genetics , School of Life Sciences, Fudan University , Shanghai , P. R. China.
b Human Phenome Institute, Fudan University , Shanghai , China.
Cell Cycle. 2018;17(16):1967-1978. doi: 10.1080/15384101.2018.1509621. Epub 2018 Sep 17.
Systemic sclerosis (SSc) is a multisystemic fibrotic disease characterized by excessive collagen deposition and extracellular matrix synthesis. Though transforming growth factor-β (TGF-β) plays a fundamental role in the pathogenesis of SSc, the mechanism by which TGF-β signaling acts in SSc remains largely unclear. Here, we showed that TGF-β type II receptor (TGFBR2) was significantly upregulated in both human SSc dermal tissues and primary fibroblasts. In fibroblasts, siRNA-induced knockdown of TGFBR2 resulted in a reduction of p-SMAD2/3 levels and reduced production of type I collagen. Additionally, functional experiments revealed that downregulation of TGFBR2 yielded an anti-growth effect on fibroblasts through inhibiting cell cycle progression. Further studies showed that miR-3606-3p could directly target the 3'-UTR of TGFBR2 and significantly decrease the levels of both TGFBR2 mRNA and protein. Furthermore, SSc dermal tissues and primary fibroblasts contain significantly reduced amounts of miR-3606-3p, and the overexpression of miR-3606-3p in fibroblasts replicates the phenotype of TGFBR2 downregulation. Collectively, our findings demonstrated that increased TGFBR2 could be responsible for the hyperactive TGF-β signaling observed in SSc. Moreover, we identified a pivotal role for miR-3606-3p in SSc, which acts, at least partly, through the attenuation of TGF-β signaling via TGFBR2 repression, suggesting that the regulation of miR-3606-3p/TGFBR2 could be a promising therapeutic target that could improve the treatment strategy for fibrosis.
系统性硬化症(SSc)是一种多系统纤维性疾病,其特征是胶原过度沉积和细胞外基质合成。尽管转化生长因子-β(TGF-β)在 SSc 的发病机制中起着基本作用,但 TGF-β信号在 SSc 中的作用机制在很大程度上仍不清楚。在这里,我们发现 TGF-β Ⅱ型受体(TGFBR2)在人类 SSc 皮肤组织和原代成纤维细胞中均显著上调。在成纤维细胞中,TGFBR2 的 siRNA 诱导敲低导致 p-SMAD2/3 水平降低和 I 型胶原产生减少。此外,功能实验表明,TGFBR2 的下调通过抑制细胞周期进程对成纤维细胞产生抗生长作用。进一步的研究表明,miR-3606-3p 可以直接靶向 TGFBR2 的 3'-UTR,并显著降低 TGFBR2 mRNA 和蛋白的水平。此外,SSc 皮肤组织和原代成纤维细胞中 miR-3606-3p 的含量明显降低,miR-3606-3p 在成纤维细胞中的过表达复制了 TGFBR2 下调的表型。总之,我们的研究结果表明,TGFBR2 的增加可能是 SSc 中观察到的 TGF-β 信号过度激活的原因。此外,我们确定了 miR-3606-3p 在 SSc 中的关键作用,它至少部分通过抑制 TGFBR2 来减弱 TGF-β 信号,表明 miR-3606-3p/TGFBR2 的调节可能是改善纤维化治疗策略的有前途的治疗靶点。