Gene Therapy and Advanced Biotech Drugs

Gene therapy and biotech drugs-based research is at an inflection point and experiencing tremendous growth. With the onset of novel therapies, powerful genomic solutions are needed to help ensure the therapy is optimized to deliver the intended end product. The course will provide the students with the advanced knowledge on the new methods for gene editing, including the last available CRISPR/Cas9 systems or the adenoviruses- or lentiviruses-associated transfection. This will be put in the context of an efficient cellular delivery, with the aim of designing nanoparticles able to carry the drug and deliver it within the desired body compartments or tissue. Also, topics like nanoaparticles bio-distribution and characterisation techniques will be addressed, especially in the context of in vivo applications. At the end of the classes, the students will learn how to reversely engineer a class of biotechnological drug, which can be designed for the treatment of a specific genetic disorder-related disease.