Amsterdam University Medical Center
Upon resolving the genetics of Gilberts syndrome and Crigler-Najjar syndrome (CNs), I have started developing novel treatments to treat inherited liver diseases. We obtained proof of concept in relevant animal models for CNs and PFIC3. In collaboration with an European consortium, including patient organizations from three European countries, a clinical trial for AAV mediated gene therapy for CNs has been started. For diseases causing liver damage I currently focus on gene repair strategies.