Study Rationale:
Inadequate understanding of how Parkinson's disease develops is a major roadblock in developing effective therapeutics and methods to diagnose the disease earlier. This project directly addresses this need by developing a unique human cellular system for the early identification and study of Parkinson's mechanisms.
Hypothesis:
The hypothesis is that cells, in particular skin fibroblasts and dopamine neurons, obtained from Parkinson's patients show specific disease-relevant features that distinguish them from cells of unaffected individuals.
Study Design:
The investigator plans to grow skin fibroblasts derived from multiple Parkinson's patients and age-matched controls and compare their specific characteristics, in terms of growth, structure and function. Likewise, she will assess dopamine neurons created from stem cells of Parkinson's and control subjects through similar readouts of growth, structure and function. The study will also examine the association between the characteristics of fibroblasts and dopamine neurons arising from the same subject.
Impact on Diagnosis/Treatment of Parkinson’s Disease:
This project will establish the utility of patient-derived fibroblasts and dopamine neurons as accurate reporters of Parkinson's status and lay the foundation for developing early diagnostic and prognostic biomarkers for Parkinson's. The study will also create a tractable human cellular model for testing the efficacy of promising drugs.
Next Steps for Development:
Successful completion of the project will support further investigations, in a larger selection of fibroblasts and dopamine neurons from different Parkinson's patients, to identify specific biomarkers relevant to Parkinson's progression and create a robust platform for future human trials.