Cellular, Molecular and Functional Approaches to Genetic Disease

The aim of the course is to highlight the different phases that characterize the study of genetic diseases to identify targets of intervention and develop novel therapeutic strategies. We will discuss about: i) identification and function of genes related to genetic disorders; ii) the use and choice of in vitro and in vivo disease models; iii) strategies to identify pathogenic mechanisms and new drug targets; iv) identification of pharmacological targets and development of new drugs; v) Animal research and preclinical studies; vi) innovative systems of "delivery" of drugs or molecules by means of viral vectors and nano-carriers; vii) clinical studies in patients. In addition, current scientific issues relating to animal testing, technological transfer of academic results, Research Integrity and the importance of research support tools (funding, peer-review process and scientific dissemination) will be addressed.

The course aims to provide students with the tools necessary to study genetic diseases. In particular, at the end of the course, the student will be able to:
1. Identify and understand the main processes that characterize the different phases of basic research and research applied to genetic diseases.
2. Apply this knowledge to choose the most suitable approaches and disease models to study the different aspects concerning the study of genetic diseases and propose new solutions.
3. Acquire autonomy and judgment on current scientific issues and / or of general interest concerning the world of research with respect to society.