Gene and cell therapeutic systems

Gene therapy vector design, generation of genetically tailor-made cells as cellular disease models, development of cellular assays, engineered cells for production.

Modern genetic engineering allows for the generation of cells tailor-made for individual applications. Cells can be engineered to replicate disease characteristics in cell culture, act as cellular test system, or maximise the production of macromolecules such as proteins, virus particles or RNA- and/or protein-loaded extracellular vesicles.

Genetic manipulation of patient cells by gene therapy vectors allows for the first time to correct the primary causes of monogenetic diseases. In the meantime, more than 20 gene therapy products have received marketing authorisation and genetically modified cells are state of the art in research and production.

The working group is involved in the development of gene therapy vectors, expression vectors and the production of genetically modified cells for production or as test systems.