Generation of Antibody and Viral tools to Advance Target Validation for Parkinson’s Disease

Parkinson’s disease is a progressive neurodegenerative disorder where the pathogenesis involves complex interaction between genetic and environmental factors. Defects in synaptic vesicle endocytosis precede dopaminergic neuron loss through an imprecisely defined mechanism. Our lab has identified molecules that are phosphorylated downstream of LRRK2 in animal models of Parkinson's disease. This project will produce antibody and AAV-based CRISPR/Cas tools against these targets which will be shared in the Parkinson's community in order to validate the role and extend the mechanistic understanding of the function of these proteins and their specific phosphorylation regulation in models of Parkinson’s disease and in patient fibroblast and PBMC cells.