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IMBA researchers find potential therapeutic approach against kidney failure

Focal segmental glomerulosclerosis (FSGS) is a severe disease that results in complete kidney failure (end-stage renal disease). If untreated or if a patient does not respond to therapy, dialysis becomes essential 2-3 times per week. While several genetic mutations are described and drug abuse or hypertension can cause FSGS, for the majority of FSGS cases the underlying cause remains elusive.

 25.04.2013

Recently, a team of researchers, amongst them Christoph Gebeshuber, postdoc at IMBA, found out that over-expression of miR-193a in podocytes (the cell type establishing the filtration barrier within the kidney glomerulus) induces FSGS in transgenic mice by down-regulating WT-1, a transcription factor essential for podocyte homeostasis. Their data showed that a huge subgroup of FSGS patients exhibits enhanced miR-193a levels. 

Preliminary experiments in mice suggest that functional inhibition of miR-193a might be a therapeutic approach in individuals with miR-193a-driven FSGS.
 

Original Publication
Gebeshuber, Kornauth, et.al. Focal segmental glomerulosclerosis is induced by microRNA-193a and its downregulation of WT1. Nat Med. 2013 Apr;19(4):481-7. doi: 10.1038/nm.3142. Epub 2013 Mar 17.