Hepatocyte-directed gene therapy for inherited disorders with liver fibrosis

Gene therapy for inherited liver disorders has undergone tremendous development. Recombinant adeno-associated viral vectors (rAAVs) are actually considered as the vector of choice for the treatment of inherited liver diseases and have recently achieved remarkable successes in clinics. Nevertheless, integrity of liver architecture is considered prerequisite for efficient gene transfer using rAAVs and patient with clinically relevant liver fibrosis are excluded from clinical trials. In our lab we develop novel gene therapy approaches to achieve clinically effective levels of liver transduction in presence of fibrosis. The "Total Award" amount indicated for this project represents the share of the funding of the Telethon Foundation for research by the Tigem institute from July 2016 until last budget year, calculated based on the size of the research group.