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.

At the end of the course, the student will acquire various types of skills to study genetic (and non) genetic diseases and critically evaluate the different phases that characterize basic research and applied research.