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Assessing the Effects of Parkin Activation in Pre-clinical Models

Study Rationale:
Protein parkin is involved in several subtypes of Parkinson's disease (PD). Loss of its activity leads to early-onset Parkinson's; therefore, increasing parkin activity may improve patient outcomes. In this study, we will use pre-clinical models with a mutation (genetic change) in parkin that improves its activity. We aim to determine if this mutation improves the outcomes in moledls following exhaustive exercise that can lead to inflammation.

Hypothesis:
We hypothesize that, unlike pre-clinical models that produce regular parkin, models that produce overactive parkin will not develop inflammation following exhaustive exercise.

Study Design:
Using a previously established research method, we will subject pre-clinical models producing overactive parkin to exhaustive exercise, collect blood samples from them and analyze the samples for cytokines, chemical signals of inflammation.

Impact on Diagnosis/Treatment of Parkinson's Disease:
Knowing whether to activate parkin, and if so, how to accomplish that, may offer new treatment strategies not only for inherited forms of Parkinson's but also potentially for PD with an unknown cause.

Next Steps for Development:
If this study is successful, we would evaluate the loss of dopaminergic neurons -- a type of brain cell affected in PD -- and the changes in movement in another pre-clinial model.


Researchers

  • Richard J. Youle, PhD

    Bethesda, MD United States


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