Objective
Our goal is to establish a successful gene therapy protocol for autonomic dominant polycentric kidney disease (ADPKD), which is the most frequent renal genetic disease (prevalence 1:1,000). 50% of ADPKD patients require renal replacement therapy by the age of 60, resulting in health care costs of 500 million Euro/year. No causal therapy for ADPKD is known. We will pursue a gene therapy strategy by establishing adequate knockout animal models, by elucidating the role of genes involved in relevant pathogenesis mechanisms and by identifying genetic modifiers affecting the severity of ADPKD. Our work will not only advance the basic knowledge on ADPKD, but it will also yield a number of candidates for gene therapy. Meanwhile, efforts will be devoted to optimising existing kidney gene transfer methods by novel delivery strategies and vectors. Finally, gene therapy experiments in animal models will set the basis for further studies in humans.
Fields of science (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques.
- medical and health sciencesmedical biotechnologygenetic engineeringgene therapy
- medical and health sciencesclinical medicinenephrologykidney diseases
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Call for proposal
Data not availableFunding Scheme
CSC - Cost-sharing contractsCoordinator
69117 HEIDELBERG
Germany