Approcci per l'identificazione di bersagli farmacologici nelle malattie genetiche

The course addresses the main phases that characterize the research path in the field of genetic
diseases, from basic research to in vivo preclinical research and the beginning of clinical trials. In
particular, through examples, we will address the main molecular, cellular and functional approaches
that allow us to understand dysfunctions and the mechanisms underlying genetic diseases and,
subsequently, identify new targets of therapeutic interest. Furthermore, current scientific issues will
be addressed regarding animal testing, the technological transfer of academic results, Research
Integrity and the importance of research support tools (funding, the peer-review process and
scientific dissemination).

The course aims to provide students with the necessary tools to study genetic diseases at all stages
of the research process, from basic research to applied research for the development of new
therapeutic strategies. At the end of the course, the student will be able to: 1. Identify and understand
the main processes that characterize the different stages of basic research and applied research on
genetic diseases. 2. Apply this knowledge to choose the most suitable approaches and disease
models to study the various aspects related to genetic disease research and propose new solutions.
Apply this knowledge to choose the most suitable approaches and disease models to study the
various aspects related to the study of genetic diseases and propose new solutions. 3. Acquire
autonomy and judgment on current scientific topics and/or general interest issues related to the world
of research with society.