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Funded Studies

In Vivo Validation of the AAV-alpha-synuclein Vectors Generated by UNC for Distribution to MJFF Supported Research Community

The Michael J. Fox Foundation would like to generate those tools and reagents that can be of greatest use to PD researchers. Working with Dr. R. Jude Samulski at The University of North Caroline at Chapel Hill Vector Core Service, MJFF is supporting the generation of wild-type alpha-synuclein and green fluorescent protein (GFP) control viral vectors in both an AAV2 and AAV5 backbone. Drs. Ron Mandel and Deniz Kirik of The University of Florida and Lund University (respectively) will work to validate these recombinant adeno-associated viral vectors in an identical manner, as reagents that can be used to model Parkinson’s disease.

Project Description:
Drs. Mandel, Kirik and others have described a method using viral vectors that over-express alpha-synuclein as a model to study mechanisms and treatments of PD. However, this model is not widely available since not every laboratory can manufacture the viral vectors required. MJFF wishes to make these valuable reagents available to the entire PD research community. In order to make sure the viral vectors can be used by everyone equally, this project will test the vectors for proper function in two independent laboratories using nearly identical techniques.

Relevance to Diagnosis/Treatment of Parkinson’s Disease:
Pre-clinical genetic models of alpha-synuclein mutations or over-expression do not induce much pathology in the nigrostriatal system. On the other hand, viral vector mediated over-expression in substantia nigra induces robust pathology including cell death and axonal abnormalities presumably associated with abnormal accumulation of alpha-synuclein. Making the viral vector model more widely available to the research community should accelerate treatment discovery especially if mechanisms of alpha-synuclein toxicity are elucidated.

Anticipated Outcome:
It is anticipated that the viral vectors will function properly as a model and become available to the PD research community.


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