The course will focus on the basic principles of gene and cell therapy, in a clinical and biological perspective. The course is aimed at providing the basic knowledge on gene transfer and gene editing (e.g. CrisprCas9) systems, based on viral and non-viral vector systems, including liposome, AAV, adenoviral, lentiviral, retroviral and herpesvirus based vectors. Untargeted and targeted gene and stem cell therapy approaches for treatment of human diseases, including bone dysplasia, cystic fibrosis, muscular dystrophy, immunodeficiencies, premature aging syndromes and cancer will be analyzed. Preclinical and clinical data will be discussed. The course will include a practical section for the development of a critical view on applied research