Ediga Harshavardhana H, Vemulapalli Chanukya P, Sontake Vishwaraj, Patel Pradeep K, Miyazaki Hikaru, Popov Dimitry, Jensen Martin B, Jegga Anil G, Huang Steven K, Englert Christoph, Schedl Andreas, Gupta Nishant, McCormack Francis X, Madala Satish K
Division of Pulmonary, Critical Care and Sleep Medicine, Department of Internal Medicine, University of Cincinnati, Cincinnati, Ohio, USA.
Gordian Biotechnology, South San Francisco, California, USA.
J Clin Invest. 2025 Jun 10;135(15). doi: 10.1172/JCI188819. eCollection 2025 Aug 1.
Idiopathic pulmonary fibrosis (IPF) is a fatal fibrotic lung disease characterized by impaired fibroblast clearance and excessive extracellular matrix (ECM) protein production. Wilms tumor 1 (WT1), a transcription factor, is selectively upregulated in IPF fibroblasts. However, the mechanisms by which WT1 contributes to fibroblast accumulation and ECM production remain unknown. Here, we investigated the heterogeneity of WT1-expressing mesenchymal cells using single-nucleus RNA-Seq of distal lung tissues from patients with IPF and control donors. WT1 was selectively upregulated in a subset of IPF fibroblasts that coexpressed several prosurvival and ECM genes. The results of both loss-of-function and gain-of-function studies were consistent with a role for WT1 as a positive regulator of prosurvival genes to impair apoptotic clearance and promote ECM production. Fibroblast-specific overexpression of WT1 augmented fibroproliferation, myofibroblast accumulation, and ECM production during bleomycin-induced pulmonary fibrosis in young and aged mice. Together, these findings suggest that targeting WT1 is a promising strategy for attenuating fibroblast expansion and ECM production during fibrogenesis.
特发性肺纤维化(IPF)是一种致命的纤维化肺部疾病,其特征在于成纤维细胞清除受损和细胞外基质(ECM)蛋白产生过多。威尔姆斯瘤1(WT1)是一种转录因子,在IPF成纤维细胞中选择性上调。然而,WT1促成纤维细胞积累和ECM产生的机制仍不清楚。在这里,我们使用来自IPF患者和对照供体的远端肺组织的单核RNA测序研究了表达WT1的间充质细胞的异质性。WT1在共表达几种促生存和ECM基因的IPF成纤维细胞亚群中选择性上调。功能丧失和功能获得研究的结果均与WT1作为促生存基因的正调控因子的作用一致,以损害凋亡清除并促进ECM产生。WT1在成纤维细胞中的特异性过表达在博来霉素诱导的年轻和老年小鼠肺纤维化过程中增强了纤维增殖、肌成纤维细胞积累和ECM产生。总之,这些发现表明,靶向WT1是在纤维化过程中减弱成纤维细胞扩张和ECM产生的一种有前景的策略。