understanding the pathophysiological mechanisms causing neurodegenerative disorders like Parkinson´s and Alzheimer´s disease. Special attention is paid to proteins of the synuclein family, since a-synuclein is a major component of aggregate structures in both diseases.

development of adeno-associated viral vector (AAV) technology, enabling us to express any kind of protein or regulatory RNA in cells refractory to conventional gene transfer methods both in vitro and in vivo. AAV vectors are also used for establishment of pre-clinical gene therapy protocols for treatment of Parkinson´s disease and other CNS disorders.

Vector tech and general applications

Gene therapy 1: MoCo deficiency

Gene therapy 2: Pharmacological control of therapeutic transgene expression

Gene therapy 3: Dual AAV for large transgenes: treatment of deafness

Synuclein research 1: pathophysiological effects on mitochondrial integrity

Synuclein research 2: pathophysiological effects on neuronal network activity