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  • Title: The podocyte as a target: cyclosporin A in the management of the nephrotic syndrome caused by WT1 mutations.
    Author: Stefanidis CJ, Querfeld U.
    Journal: Eur J Pediatr; 2011 Nov; 170(11):1377-83. PubMed ID: 21298518.
    Abstract:
    Children with steroid-resistant nephrotic syndrome secondary to WT1-associated glomerulopathies (WT1-GP) were considered unresponsive to cyclosporin A (CsA). This assumption is challenged by the findings of recent studies. The patients of these studies had different types of WT1 mutations and varying clinical presentations. However, all of them were of young age and the favourable response to CsA might be the result of treatment at an early stage of the disease. The additional administration of angiotensin-converting enzyme inhibitors may have contributed to the positive outcome. We review recent data on the role of WT1 in the development of WT1-GP and discuss putative therapeutic targets explaining the therapeutic effect of CsA.
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