Gene therapy and viral vectors: technologies and applications

This activity introduces the principles of gene therapy and its main therapeutic applications. It also covers molecular targets and genome modification techniques.

This activity presents the types of vectors used in gene therapy (viral/non-viral vectors, integrative/non-integrative vectors), their functions, advantages and limitations. This activity also introduces some innovative prospects for the development of gene therapy vectors.

This activity covers the key stages in the viral vector biomanufacturing process, based on the example of AAV and lentiviral vectors, with a particular focus on raw materials, equipment and industrial challenges.