Saha Dyuti, Annadorai Ravi Kiran, Thannimangalath Sujaya, Shroff Neha P, Kataria Sunny, Dam Binita, Dutta Abhik, Hegde Akshay, Hiwale Ankita, Ravula Venkatesh, Saha Shagnik, Minikumari Rahulan Lekshmi, Nigam Neha, Singh Neha, Agarwal Vikas, Vemula Praveen K, Jamora Colin
IFOM-inStem Joint Research Laboratory, Institute for Stem Cell Science and Regenerative Medicine, Bangalore, India.
Department of Biology, Manipal Academy of Higher Education, Manipal, India.
J Clin Invest. 2025 Jun 20;135(15). doi: 10.1172/JCI177123. eCollection 2025 Aug 1.
Vasculopathy is a common hallmark of various fibrotic disorders, including systemic sclerosis (SSc), yet its underlying etiology and contribution to fibrogenesis remain ill defined. In SSc, the vasculopathy typically precedes the onset of fibrosis, and we observed that this phenomenon is recapitulated in the Snail transgenic mouse model of SSc. The vascular anomalies manifest as deformed vessels, endothelial cell dysfunction, and vascular leakage. Our investigation into the underlying mechanism of this phenotype revealed that angiopoietin-like protein 2 (ANGPTL2), secreted by the Snail transgenic keratinocytes, is a principal driver of fibrotic vasculopathy. In endothelial cells, ANGPTL2 upregulates profibrotic genes, downregulates various junctional proteins, and prompts the acquisition of mesenchymal characteristics. Inhibiting endothelial cell junctional instability and, consequently, vascular leakage with a synthetic analog of the microbial metabolite Urolithin A (UAS03) effectively mitigated the vasculopathy and inhibited fibrogenesis. Thus, ANGPTL2 is a promising early biomarker of the disease, and inhibiting the vasculopathy-inducing effects of this protein with agents such as UAS03 presents an appealing therapeutic avenue to reduce disease severity. These insights hold the potential to revolutionize the approach to treatment of fibrotic diseases by targeting vascular defects.
血管病变是包括系统性硬化症(SSc)在内的各种纤维化疾病的常见特征,但其潜在病因及其对纤维化形成的作用仍不清楚。在系统性硬化症中,血管病变通常先于纤维化出现,并且我们观察到这种现象在系统性硬化症的Snail转基因小鼠模型中也有重现。血管异常表现为血管变形、内皮细胞功能障碍和血管渗漏。我们对这种表型潜在机制的研究表明,Snail转基因角质形成细胞分泌的血管生成素样蛋白2(ANGPTL2)是纤维化血管病变的主要驱动因素。在内皮细胞中,ANGPTL2上调促纤维化基因,下调各种连接蛋白,并促使细胞获得间充质特征。用微生物代谢产物尿石素A(UAS03)的合成类似物抑制内皮细胞连接不稳定以及由此导致的血管渗漏,可有效减轻血管病变并抑制纤维化形成。因此,ANGPTL2是一种很有前景的疾病早期生物标志物,用UAS03等药物抑制该蛋白的血管病变诱导作用,为减轻疾病严重程度提供了一条有吸引力的治疗途径。这些见解有可能通过针对血管缺陷彻底改变纤维化疾病的治疗方法。