Feasibility of preventing of renal cyst formation in utero studied in mice

What is it about?

Renal cyst formation begins in uetero and is strongly associated with pathogenesis of polycystic kidney disease (PKD), especially in recessive forms of PKD. To investigate the feasibility of preventing cyst formation in utero we used rapamycin to treat fetuses (while still in utero) that were developing renal cysts. We show in this publication that it is indeed feasible to inhibit cyst growth in utero, although rapamycin may not be the most suitable agent to use for this purpose.

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Why is it important?

There are presently no treatments available to prevent or reverse cyst formation in patients with polycystic kidney disease (PKD). Prevention of cyst formation when cysts are forming (eg in utero, when the cysts are still very small) might help to prevent full blown cyst formation and therefore help to delay features of later stages in polycystic kidney disease (PKD), which might be helpful in preventing onset of renal failure in patients with PKD. We show this is feasible using mice as a model.