Lecturer: Prof. Dr.-Ing. Jørgen Magnus (RWTH Aachen)
This lecture describes how adeno associated virus (AAVs) are used as vectors to transport genetic material into cells in an in-vivo gene therapy. It starts by giving a general introduction to gene therapy before it shows the AAV biology and the molecular design of the recombinant AAV vectors. The three most common expression systems used for the production of AAVs are then described highlighting the pros and cons, before the manufacturing process including the purification is explained in detail. Finally, the gene therapy LUXTURNA is used as an example to show the mode of action and the development of a successful gene therapy based on AAVs.