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Funded Studies

rAAV-ribozyme Mediated Parkin Knockdown as a Pre-clinical Model of Parkinson's Disease

Parkinson's disease is characterized by a series of debilitating motor abnormalities that worsen over time. The motor symptoms of Parkinson's disease are largely the result of the specific progressive degeneration of dopamine-producing neurons in the substantia nigra located in the midbrain. Several novel treatment strategies have recently been described that have proven effective in aborting dopamine cell death in toxin-based animal models of Parkinson's disease. These toxin based Parkinson's models faithfully recapitulate the specific cell death of nigral dopamine neurons. However, whether the precise cellular mechanisms of this toxin-induced dopamine cell death are similar to those causing the degeneration observed in Parkinson's disease is unknown. Thus, it is unclear whether these novel treatment strategies are relevant to human Parkinson's disease. Recently, several familial forms of Parkinson's disease have been described with mutations in specific genes. We propose to attempt to use the viral vector, recombinant adeno-associated virus to shut down the production of one of the familial Parkinson's disease gene products, parkin, specifically in the substantia nigra of an animal in order to create a new animal model of Parkinson's disease. The proposed studies take advantage of the serendipitous fact that recombinant adeno-associated viral vectors specifically genetically engineer nigral dopamine neurons at very high efficiency. If successful in the animal, this strategy has the advantage that it can be transferred to other animal species for use in studying novel treatment strategies for Parkinson's disease.


  • Ronald J. Mandel, PhD

    Gainesville, FL United States

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