Translational therapeutics: Therapeutic endpoints and diagnostic biomarker for advanced therapy medicinal products (ATMP) against heart failure

To enable the design of meaningful clinical trial protocols for future heart failure gene therapy programs, this project aims to determine optimized parameters of therapeutic efficacy (work package 1), viral vectors (work package 2) and predictive biomarkers (work package 3) for DNA-based therapeutic targets/ATMP against chronic heart failure such as S100A1 and others. In addition, this project seeks development of disease- (work package 4) and ligand-controlled (work package 5) gene expression systems suitable for the human-sized heart to overcome limitations of currently used constitutive systems.