Programme
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