Centre for Complement and Inflammation Research, Imperial College, London, United Kingdom.
J Am Soc Nephrol. 2013 Jan;24(1):43-52. doi: 10.1681/ASN.2012060571. Epub 2012 Nov 26.
Complement factor H (CFH) is a negative regulator of the alternative pathway of complement, and properdin is the sole positive regulator. CFH-deficient mice (CFH(-/-)) develop uncontrolled C3 activation and spontaneous renal disease characterized by accumulation of C3 along the glomerular basement membrane, but the role of properdin in the pathophysiology is unknown. Here, we studied mice deficient in both CFH and properdin (CFH(-/-).P(-/-)). Although CFH(-/-) mice had plasma depleted of both C3 and C5, CFH(-/-).P(-/-) animals exhibited depletion of C3 predominantly, recapitulating the plasma complement profile observed in humans with properdin-independent C3 nephritic factors. Glomerular inflammation, thickening of the capillary wall, and glomerular C3 staining were significantly increased in CFH(-/-).P(-/-) compared with CFH(-/-) mice. We previously reported that exogenous CFH ameliorates C3 staining of the glomerular basement membrane and triggers the appearance of mesangial C3 deposits in CFH(-/-) mice; here, we show that these effects require properdin. In summary, during uncontrolled activation of C3 driven by complete CFH deficiency, properdin influences the intraglomerular localization of C3, suggesting that therapeutic inhibition of properdin would be detrimental in this setting.
补体因子 H (CFH) 是补体替代途径的负调节剂,而 properdin 是唯一的正调节剂。CFH 缺陷型小鼠 (CFH(-/-)) 会出现不受控制的 C3 激活和自发性肾脏疾病,其特征是 C3 在肾小球基底膜处积累,但 properdin 在病理生理学中的作用尚不清楚。在这里,我们研究了同时缺乏 CFH 和 properdin 的小鼠 (CFH(-/-).P(-/-))。尽管 CFH(-/-) 小鼠的血浆中耗尽了 C3 和 C5,但 CFH(-/-).P(-/-) 动物主要耗尽了 C3,再现了在具有 properdin 非依赖性 C3 肾炎因子的人类中观察到的血浆补体谱。与 CFH(-/-) 小鼠相比,CFH(-/-).P(-/-) 动物的肾小球炎症、毛细血管壁增厚和肾小球 C3 染色显著增加。我们之前报道过外源性 CFH 可改善肾小球基底膜的 C3 染色,并在 CFH(-/-) 小鼠中引发系膜 C3 沉积;在这里,我们表明这些效应需要 properdin。总之,在完全缺乏 CFH 导致的不受控制的 C3 激活期间,properdin 影响 C3 在肾小球内的定位,表明在这种情况下抑制 properdin 会产生不良影响。