Objective/Rationale:
A major hurdle in the development of drugs for Parkinson's disease (PD) is the lack of accurate animal models for the disease. We describe new animal models that harbor defects in an important cellular pathway and display features of PD. Such models allow for the testing of novel drugs for PD.
Project Description:
In this project, two new animal models are described, based on alterations in an important cell mechanism that regulates the function and survival of dopamine neurons – the cells that are lost in PD. These models will be used to test novel therapeutics for PD.
Relevance to Diagnosis/Treatment of Parkinson’s Disease:
We focus on the analysis of therapeutics that are already well described for other disorders and could therefore be quickly applied to PD, if effective. Such therapeutics may alter the course of PD, and no currently available therapeutics are effective in altering disease course.
Anticipated Outcome:
We will test a total of four compounds in this proposal. If a positive outcome is observed, we will proceed with safety testing to initiate clinical trials.