Marszal Joanna, Saleem Moin A
Academic and Children's Renal Unit, University of Bristol, Southmead Hospital, Bristol, UK.
Nephron Exp Nephrol. 2006;104(1):e1-5. doi: 10.1159/000093259. Epub 2006 May 11.
Focal segmental glomerulosclerosis (FSGS) is a devastating form of nephrotic syndrome, often leading to end-stage renal failure after the failure of a succession of highly toxic therapies. It has long been thought to be caused by a circulating factor(s) that may be produced by cells of the immune system. Much research has focused on identifying such factor(s), including the development of a promising in vitro assay, which estimates glomerular permeability based on the swelling of isolated glomeruli in response to patients' plasma. This assay has also been used as the basis of testing plasma fractions for permeability activity, with no specific factor yet identified. Other studies have attempted to replicate proteinuria in whole animals, by injecting plasma or plasma fractions from focal segmental glomerulosclerosis patients, with inconsistent results. More recently there has been evidence that there may be either inhibitory or missing factor(s) in plasma, with respect to permeability. An additional major biological advance is a growing appreciation of the podocyte as the target cell in this disease, and an understanding of the key molecules involved. Putting together this knowledge, with the latest technological advances in protein identification, provides promising avenues towards finally solving the basis of this enigmatic disease.
局灶节段性肾小球硬化（FSGS）是一种严重的肾病综合征形式，在一系列高毒性治疗失败后常导致终末期肾衰竭。长期以来，人们一直认为它是由免疫系统细胞可能产生的一种循环因子引起的。许多研究都集中在确定这种因子，包括开发一种有前景的体外检测方法，该方法根据分离的肾小球对患者血浆的反应肿胀来估计肾小球通透性。该检测方法也被用作检测血浆组分通透性活性的基础，但尚未确定具体因子。其他研究试图通过注射局灶节段性肾小球硬化患者的血浆或血浆组分在全动物中复制蛋白尿，但结果不一致。最近有证据表明，就通透性而言，血浆中可能存在抑制性或缺失性因子。另一个主要的生物学进展是越来越认识到足细胞是这种疾病的靶细胞，并了解其中涉及的关键分子。将这些知识与蛋白质鉴定的最新技术进展结合起来，为最终解决这种神秘疾病的病因提供了有希望的途径。
