Tsai I-Jung, Chou Chia-Hung, Yang Yao-Hsu, Lin Wei-Chou, Lin Yen-Hung, Chow Lu-Ping, Lee Hsiao-Hui, Kao Pei-Gang, Liau Wan-Ting, Jou Tzuu-Shuh, Tsau Yong-Kwei
Department of Pediatrics, National Taiwan University Hospital and National Taiwan University College of Medicine, Taipei, Taiwan.
Cell Mol Life Sci. 2015 Aug;72(16):3157-71. doi: 10.1007/s00018-015-1888-0. Epub 2015 Mar 20.
Childhood nephrotic syndrome is mainly caused by minimal change disease which is named because only subtle ultrastructural alteration could be observed at electron microscopic level in the pathological kidney. Glomerular podocytes are presumed to be the target cells whose protein sieving capability is compromised by a yet unidentified permeability perturbing factor. In a cohort of children with non-hereditary idiopathic nephrotic syndrome, we found the complement fragment C5a was elevated in their sera during active disease. Administration of recombinant C5a induced profound proteinuria and minimal change nephrotic syndrome in mice. Purified glomerular endothelial cells, instead of podocytes, were demonstrated to be responsible for the proteinuric effect elicited by C5a. Further studies depicted a signaling pathway involving Rho/Rho-associated kinase/myosin activation leading to endothelial cell contraction and cell adhesion complex breakdown. Significantly, application of Rho-associated kinase inhibitor, Y27632, prevented the protein leaking effects observed in both C5a-treated purified endothelial cells and mice. Taken together, our study identifies a previously unknown mechanism underlying nephrotic syndrome and provides a new insight toward identifying Rho-associated kinase inhibition as an alternative therapeutic option for nephrotic syndrome.
儿童肾病综合征主要由微小病变病引起,该病得名于在病理状态下的肾脏中,电子显微镜水平仅能观察到细微的超微结构改变。肾小球足细胞被认为是靶细胞,其蛋白质滤过能力因一种尚未明确的通透性干扰因子而受损。在一组非遗传性特发性肾病综合征患儿中,我们发现补体片段C5a在疾病活动期血清中升高。给小鼠注射重组C5a可诱导严重蛋白尿和微小病变肾病综合征。研究表明,纯化的肾小球内皮细胞而非足细胞,是C5a引发蛋白尿效应的原因。进一步研究描绘了一条涉及Rho/ Rho相关激酶/肌球蛋白激活的信号通路,该通路导致内皮细胞收缩和细胞黏附复合物分解。值得注意的是,应用Rho相关激酶抑制剂Y27632可防止在C5a处理的纯化内皮细胞和小鼠中观察到的蛋白质渗漏效应。综上所述,我们的研究确定了一种先前未知的肾病综合征发病机制,并为将Rho相关激酶抑制作为肾病综合征的替代治疗选择提供了新的见解。