Stem cells and genetic diseases

The course focusses on the steps that characterize the study of genetic diseases in order to identify new therapeutic strategies. The main objectives of the course are: i) to study the function of genes related to diseases; ii) to analyze in-vitro models used to study genetic disease with a specific focus on those based on stem cells; iii) to identify the methodological strategies to identify pathogenic mechanisms; iv) to describe pre-clinical studies in animal models, clinical studies, and validation of new drug targets; iv ) to describe new therapeutic approaches based on gene therapy and stem cells.

At the end of the course students will have acquired knowledge about i) molecular tools and models to study disease genes; ii) strategies to identify and validate genetic-driven druggable targets; iii) the use of drug targets in drug screening assays; iv) the use of stem cells for understanding and treating genetic disorders; v) gene and cell-based therapies.