Description of current research activities within the group

Novel genetic models of neurodegenerative diseases We are using recombinant adeno-associated viral (rAAV) vectors to overexpress disease-causing proteins in the brain of small laboratory animals (rats and mice) and in large animals (pigs and non-human primates) in order to develop better models for neurodegenerative diseases and test hypotheses. Photo: Courtesy of Tomas Björklund

Gene therapy for Parkinson's disease

The BRAINS unit is working closely with internationally leading teams that aim to explain important factors contributing to disease onset and progresseion to develop the rAAV vectors for delivery of therapeutic genes to the brain. This gene therapy approach involves intrastriatal expression of glial cell line-derived neurotrophic factor (GDNF) for neuroprotection, on one hand, and expression of the DOPA synthesizing enzymes, tyrosine hydroxylase (TH) and its co-factor enzyme GTP cyclohydrolase-1 (GCH1), for neurorestoration on the other. Both approaches provide functional recovery in animal models and are currently being developed as novel treatment approaches towards early clinical trials.

Experimental neuroimaging program

Molecular and metabolic imaging techniques have a significant impact on medical research. Over the last few years magnetic resonance spectroscopy (MRS) and nuclear dynamic polarization (DNP) technologies were developed to an extent that allows for questions with high impact in biomedical sciences to be effectively addressed. Similarly, highly specialized radioactive positron emission tomography (PET) tracers are now utilized with high spatial and temporal resolution for disease-related research in animals. The BRAINS group has recently employed several research projects that combine their expertise in disease related translational research with these novel in vivo imaging tools to address challenging issues in development of therapies for brain diseases.

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