New therapies on the basis of RNA molecules

GO-Bio 3 – Prof. Dr. Gunther Hartmann – Institute of Clinical Chemistry and Pharmacology, University Hospital Bonn

Stilisierte Dartsellung eines DNA-Strangs

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Recipient: University Hospital Bonn
Funding: GO-Bio Phase I (01.04.2010 - 30.06.2014, 1.604.190 Euro)


Nucleic acids, which carry genetic information in the form of either RNA (ribonucleic acid) or DNA (deoxyribonucleic acid), are essential substances for organisms. Today, nucleic acids are gaining in importance as a novel form of immunological therapy. Gunther Hartmann has been working in this area for some years. His work focuses on how virus genomes – often in the form of RNA – are detected by human immune defences. Within the scope of his BioFuture funding, he has been clarifying this issue at the molecular level. Following on from these findings, Hartmann’s team has developed specific immunostimulatory RNA molecules that mimic viral infections and activate specific immune responses, thus stimulating the body’s own defence mechanisms.

This new approach could be put to use in specially targeted therapies. Furthermore, the researchers have found a way of combining these RNA molecules with the properties of RNA interference. Thereby, they are hoping to achieve two different functions: stimulating the immune system and switching of specific genes that are also critical for the survival of cancer cells. Both therapeutic approaches have already been successfully tested in animal models. In their GO-Bio project, Hartmann and his research team want to develop the RNA molecules up to clinical testing before applying them as therapeutics against viral infections, cancers and autoimmune diseases. In the long term, and following the first successful validation through clinical trials, it is hoped to found a company that would out-license possible drug candidates to biotech and pharmaceutical companies.