Cell biology and technology

General objectives - The main objective of the course is to provide students with the tools to understand the main approaches of gene and cell therapy through a thorough study of microbial vectors in use both in basic research and in clinical trials. Particular attention is devoted to the development process of first-second- and third-generation vectors and to the methodological aspects for the production of a therapeutic vector. Finally, the ethical issues that derive from the use of genetic material for therapeutic purposes will be considered. Specific objectives - Knowledge and ability to understand. The specific objectives of the course include: • Understand and understand the process that from the knowledge of the molecular mechanisms underlying the functionality of specific genetic elements (viruses, chromosomes, plasmids, transposons) leads to the development of vectors for the transfer of genes / sequences in mammalian cells. • Know and understand the methods and models used to test the safety of the carriers and the related production processes. Ability to apply knowledge and understanding. The course provides the tools that allow the student to select specific microbial elements, such as plasmids, transposons and viruses, for the transfer of genetic material in vitro and in vivo, even considering the specific experimental / therapeutic needs; select the most appropriate engineering techniques for gene transfer elements; to evaluate host responses to gene transfer; apply the different methods of molecular and cellular investigation for the study of the different production phases of gene transfer systems. Making judgments: The student, through the study of the sector literature, the analysis of experimental data present in public databases, the carrying out of practical laboratory tests, acquires an autonomous ability to interpret experimental data and to place them in the framework of knowledge already acquired. It will also be able to evaluate ethical issues. Communication skills: the student acquires the use of appropriate scientific terminology to communicate the concepts underlying gene therapy, the use of means of investigation, production and the real issues of ethical nature, both to a lay public and experts . Learning skills: the ability to learn is stimulated by the continuous reference, in all the activities of the course, to the knowledge acquired and to the methodologies used for their progress. At the end of the course the students will have learned the genetic mechanisms underlying the most commonly treated pathologies with medicaments of genetic origin, the construction, production and use of genetic medications