Article
Successful treatment of steroid-resistant nephrotic syndrome associated with WT1 mutations.
Pediatric nephrology (Berlin, Germany) - 1 Jul 2010
Gellermann Jutta, Stefanidis Constantinos J, Mitsioni Andromachi, Querfeld Uwe
Abstract excerpt
The Wilms' tumor suppressor gene 1 (WT1) encodes a transcription factor involved in kidney and gonadal development. WT1 is also a key regulator of podocyte functions and mutations have been found in a small percentage of children with isolated or syndromal steroid-resistant nephrotic syndrome. It is commonly assumed that the nephrotic syndrome (NS) in patients with WT1 mutations is unresponsive to therapy and...
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