In the nucleus of human cells, double strands of deoxyribonucleic acid (DNA) store the genetic information. Usually they form a double helix structure. By cleverly choosing the sequence of the individual DNA bases, however, completely different shapes can be rationally designed. In analogy to the Japanese art of folding paper, this technique is called DNA origami. In his project named GENESHUTTLE, Prof. Hendrik Dietz aims at building virus-like objects from synthetic DNA. These assemblies should be equipped in such a way that, like real viruses, they can recognize target cells and deliver genetic material into the nucleus of these cells. Such synthetic gene delivery systems could on the one hand be used as a model systems for the investigation of viral infection processes. On the other hand, they would be potentially superior to synthetic vectors and would offer new technical possibilities for future gene therapies.

Hendrik Dietz heads the Chair of Biomolecular Nanotechnology. His research has already been founded with an ERC Starting Grant and an ERC Consolidator Grant. In 2016 he received the Leibniz Prize, the highest scientific distinction of the German Research Foundation.

Heart failure is one of the most common causes of death in Europe and often results from a weak pumping action due to genetic mutations. In the Cor-Edit-P project, Prof. Christian Kupatt wants to use CRISPR-Cas9 genome editing as therapy for these congenital conditions for which this has never been a specific treatment. He has developed a tailor-made method that uses special adeno-associated viruses (ADVs) to gain access to the cells in order to modify the relevant genome segment. He has already used this method to treat Duchenne muscular dystrophy, a hereditary disease, in pigs. Alternatively, genetically corrected cardiac precursor cells will be placed in the weakened heart muscle to help it maintain a healthier pumping action. The goal of the Cor-Edit-P project is to correct the genetic causes of cardiac weakness to optimize the heart function, reduce the risk of life-threatening cardiac arrhythmia and thus offer patients greater life expectancy and improved quality of life.

Prof. Christian Kupatt is an executive senior physician at the Clinic and Polyclinic for Internal Medicine I at the TUM university hospital Klinikum rechts der Isar.

Nerve cells are surrounded by a fatty myelin layer, or sheath, that increases their ability to transmit electrical impulses, among other functions. Myelin is produced by cells known as glial cells that perform many important tasks related to nervous system functions. In his NETWORK project, Prof. Mikael Simons will study the networking of different types of glial cells. He suspects that these cells communicate using lipoproteins, for example to deal with the natural ageing processes of the myelin sheath. With his team he plans to investigate the lipoproteins more closely to find out how they help to protect the myelin sheath. Prof. Simons is also interested in the role of lipoproteins in neurodegenerative or neuroinflammatory diseases such as multiple sclerosis, in which the myelin sheath is destroyed through inflammation processes.

Mikael Simons is Professor for Molecular Neurobiology. His research has previously been funded with an ERC Starting Grant and a Consolidator Grant.

Although breathing is an essential process for humans and lung diseases are one of the leading causes of death, there are still a number of unsolved mysteries regarding vital processes in the lungs. The main reason for this is the lack of measurement methods or imaging techniques in crucial regions of the lung. Computer models of the lung are opening up new perspectives. While some of these models are already promising on their way to clinical use, even the best models still suffer from severe limitations. With the BREATHE project, Prof. Wolfgang A. Wall aims to develop unprecedented comprehensive computational models of the respiratory system. With crucial new developments in the field of high-performance computing, coupled tissue and fluid mechanics and the combination of machine learning and physical modeling, important open questions in biomedical research will be answered and, based on available heterogeneous patient data, decisive steps towards personalized medicine will be taken.

Wolfgang A. Wall is Professor for Computational Mechanics.