Horsch Marion, Beckers Johannes, Fuchs Helmut, Gailus-Durner Valérie, Hrabě de Angelis Martin, Rathkolb Birgit, Wolf Eckhard, Aigner Bernhard, Kemter Elisabeth
German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München GmbH, German Research Center for Environmental Health, Neuherberg, Germany.
German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München GmbH, German Research Center for Environmental Health, Neuherberg, Germany; German Center for Diabetes Research (DZD), Neuherberg, Germany; Experimental Genetics, Center of Life and Food Sciences Weihenstephan, Technische Universität München, Freising-Weihenstephan, Germany.
PLoS One. 2014 Nov 19;9(11):e113125. doi: 10.1371/journal.pone.0113125. eCollection 2014.
Uromodulin-associated kidney disease (UAKD) is a hereditary progressive renal disease which can lead to renal failure and requires renal replacement therapy. UAKD belongs to the endoplasmic reticulum storage diseases due to maturation defect of mutant uromodulin and its retention in the enlarged endoplasmic reticulum in the cells of the thick ascending limb of Henle's loop (TALH). Dysfunction of TALH represents the key pathogenic mechanism of UAKD causing the clinical symptoms of this disease. However, the molecular alterations underlying UAKD are not well understood. In this study, transcriptome profiling of whole kidneys of two mouse models of UAKD, UmodA227T and UmodC93F, was performed. Genes differentially abundant in UAKD affected kidneys of both Umod mutant lines at different disease stages were identified and verified by RT-qPCR. Additionally, differential protein abundances of SCD1 and ANGPTL7 were validated by immunohistochemistry and Western blot analysis. ANGPTL7 expression was down-regulated in TALH cells of Umod mutant mice which is the site of the mutant uromodulin maturation defect. SCD1 was expressed selectively in the S3 segment of proximal tubule cells, and SCD1 abundance was increased in UAKD affected kidneys. This finding demonstrates that a cross talk between two functionally distinct tubular segments of the kidney, the TALH segment and the S3 segment of proximal tubule, exists.
尿调节蛋白相关肾病(UAKD)是一种遗传性进行性肾病,可导致肾衰竭,需要肾脏替代治疗。由于突变型尿调节蛋白成熟缺陷及其在亨氏袢厚升支(TALH)细胞内质网扩张中的滞留,UAKD属于内质网储存疾病。TALH功能障碍是UAKD导致该疾病临床症状的关键致病机制。然而,UAKD潜在的分子改变尚未完全明确。在本研究中,对两种UAKD小鼠模型UmodA227T和UmodC93F的全肾进行了转录组分析。通过RT-qPCR鉴定并验证了在不同疾病阶段UAKD影响的两个Umod突变系肾脏中差异丰富的基因。此外,通过免疫组织化学和蛋白质印迹分析验证了SCD1和ANGPTL7的差异蛋白丰度。ANGPTL7在Umod突变小鼠的TALH细胞中表达下调,而TALH细胞是突变型尿调节蛋白成熟缺陷的部位。SCD1在近端小管细胞的S3段选择性表达,且在UAKD影响的肾脏中SCD1丰度增加。这一发现表明,肾脏中两个功能不同的肾小管节段,即TALH节段和近端小管的S3节段之间存在相互作用。