Study Rationale:
This study aims to produce data that will help to understand the molecular processes and networks underlying Parkinson’s disease (PD). In this study, we will use cells from people who participated in the Parkinson’s Progression Markers Initiative (PPMI), our large-scale, international biomarker study.
Hypothesis:
Research approaches including complex genetics will generate critical information about the molecular processes underlying PD.
Study Design:
Cells donated by PPMI participants will be grown into nerve cells similar to those found in the brain. Characterization of the molecules that make up these cells will provide a map of interactions of PD-linked genetic changes at the cellular and molecular levels and explain how these changes cause disease.
Impact on Diagnosis/Treatment of Parkinson’s disease:
This work has the potential to improve diagnosis by defining subtypes of disease with different molecular profiles. Most importantly, this work has the potential to identify key networks involved in disease and to discover feasible targets for therapeutic intervention.
Next Steps for Development:
The clinical application of these results will rely on the testing of potential therapeutics for their ability to act upon the identified targets. Notably, this system can be used to test whether the intervention is on target – and whether there are any unanticipated or undesirable side effects.