220. AAV Mediated Gene Transfer of Smad7 in Different Models of Cystic Kidney Diseases

Cystic diseases of the kidney (CDK) with consecutive interstitial fibrosis represent the most frequent hereditary causes of end-stage renal failure in children and adults. Up to now no causal treatment for any of them is available in humans. It is shown that the TGF-|[beta]| pathway is playing a major role in the development of interstitial fibrosis and an overexpression of Smad7, the negative inhibitor of TGF-|[beta]|, is able to prevent fibrosis. Therefore we want to analyse the effect of a long term expression of Smad7 on renal function and interstitial fibrosis in our CDK animal models.