Lecturer: Prof. Dr. Hannes Klump (RWTH Aachen Medical School)
Following a brief definition of the term “Gene Therapy,” the lecture provides an overview of the vector systems commonly employed for gene transfer into target cells. The remaining lecture focuses primarily on retroviral vectors, with particular attention given to the retroviral replication cycle and the manner in which this knowledge has been leveraged to derive safer vector designs. Owing to their ability to integrate into the host genome, the distinct integration patterns of gammaretroviral and lentiviral vectors are examined, together with the assay systems used to characterize these patterns and to assess vector safety. The lecture concludes with a discussion of strategies aimed at enhancing their safety profile, as well as examples of the application of such vectors in the development of novel cell-based therapies.