Anders Björklund Professor, MD/PhD
Current research in our group is focussed on the use of neural stem cells and viral vector-mediated gene transfer for neuroprotection and brain repair, with the aim to develop new therapeutic approaches for Parkinson´s disease and other neurodegenerative disorders.
Stem cells and gene delivery have opened new possibilities for restoration of function in nervous system diseases by promotion of the brains capacity to repair itself. Our laboratory has pioneered the use of cell transplantation and recombinant viral vectors for neurodegenerative disease. Over the last decade our work has been focused on the development of novel restorative therapies for Parkinsons disease, based on the use of neural stem and progenitor cells for brain repair and the application of techniques for in vivo gene delivery to the brain.
(1) We use cells obtained from rat and human embryos to restore dopamine neurotransmission in rats with lesions of the nigrostriatal dopamine system.
(2) We use neural stem- and progenitor cells for cell transplantation in the brain. Cells with stem cell-like properties, expanded in vitro as neurospheres under the influence of growth factors, show extensive migration and site?specific neuronal differentiation after transplantation to neurogenic regions of the adult brain, suggesting that grafted progenitors can differentiate along multiple phenotypic pathways in vivo.
(3) We use recombinant lentiviral and adenoassociated viral (AAV) vectors to deliver neurotrophic factors and neurotransmitter-synthesising enzymes (TH and AADC) to restore dopmine neurotransmission in the rat Parkinson model. In particular, we have shown that intrastriatal and/or intranigral delivery of GDNF, using viral vectors, can provide complete protection, and functional restoration, of the nigrostriatal dopamine system.
(4) We use rAAV vectors to overexpress alpha-synuclein in substantia nigra in adult rats. Using this approach we have developed a new rat model of Parkinson’s disease which reproduces many of the neuropathological and functional features of the human disease.