Understanding mechanisms of disease and development of new therapeutic strategies for neurodegenerative diseases is a challenging task and constitutes an area of major importance for the neuroscience community.
The BRAINS group contributes to this task with its expertise in studies utilizing animal models of human diseases. A recent example is development of a new animal model for Parkinson's disease based on targeted overexpression of alpha-synuclein in the brain. This model provided a novel tool for studies of progressive neuropathology and cell death in the nigrostriatal system, similar to that seen in Parkinson patients.
Recombinant viral vectors are very powerful tools for in vivo gene therapy in the CNS. The BRAINS group puts a special emphasis on development of viral vectors toward clinical applications as tools to deliver therapeutic molecules to the brain.