Cellular and animal models for understanding complex genetic disorders

Undestanding the mechanisms underlying disease at the levels of molecules, cells, and neuronal circuits is essential for developing more effective therapies for neurological disorders. In particular, neurodevelopmental alterations may already establish disease risk at early life stages. We use molecular and cellular assays to gain insight into disease mechanisms. We also generate human organoid and animal models carrying disease-associated genetic variants using CRISPR/Cas9 genome engineering. For generating and characterizing animal models, we work closely with the Institute of Developmental Genetics and the German Mouse Clinic, both at the Helmholtz Zentrum München.

Moreover, the gene-environment interaction is crucial for disease manifestation and represents a potentially fruitful avenue for prevention strategies. We have a particular focus on modeling changes in internal environment. We also explore lifestyle and nutritional factors which can exacerbate neurological disorders.