Novel approaches to gene delivery

Development of protein-based DNA transfection agents and chemical transfection enhancers

Gene therapy holds the potential to revolutionize medicine. However, the efficient and safe delivery of DNA molecules into the nucleus of mammalian cells still poses a big challenge. Most therapies that are on the market or in clinical trials make use of viruses, which show great transfection efficacies but suffer from severe side effects. Non-viral systems, on the other hand, are more versatile, easier to produce and safer, but generally lack efficiency.

In this project, we follow two strategies to increase the transfection efficiency of non-viral vectors. First, we develop new transfection agents based only on recombinant proteins. This allows us to freely combine and modify the properties of existing proteins, to form stable DNA particles that can eventually overcome the many hurdles on their journey to the nucleus. Second, we develop screening assays to find inhibitors against specific cellular DNA defense systems. These will eventually be used in combination with the currently available transfection agents to fully exploit their potential.

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