Cell Therapy and Gene Silencing

The course is organized in three sections including single bench laboratory and practical activities.
The Cell-based Technology section (2 CFU) will cover the aspects relative to the use of cellular models in biomedical research. These include aspects of cell culture manipulation, differences between immortalised and primary cells, stems cells, iPSCs, CAR T cells, methods of transfection and protein production.
The Viral Vectors for Gene Delivery section (2 CFU) will cover the aspects relative to the use of viral gene transfer/gene therapy including viral replication and gene expression strategies, retroviral and lentiviral vectors and applications, adenoviral vectors and Adenovirus-Associated virus (AAV) vectors and relative applications, innate immune response to viral vectors, the use of viral vectors for gene editing, RNAi, epigenetic silencing and genetic vaccines.
The Pharmacological aspects of Cell Therapy and Gene Silencing section (1 CFU) will cover some examples of pharmacological use and outcomes on human diseases. In details, discussion of pathological mechanisms, pharmacological strategies and clinical trials of gene therapy (in vivo and ex vivo), gene silencing approaches (siRNA and ASO) and epidrugs.

Practical sections are organized in two parts: the first will include demonstrations on cell culture, transfection, protein production and localization (1 CFU); the second part will deal with omics application by the use of online software for the analysis and interpretations of biological implications of gene therapy approaches and exercise of clinical pharmacology (1 CFU).