Study Rationale: This effort aims to produce foundational data that will provide immediate insight and a resource for understanding the molecular basis of Parkinson disease (PD). The work, conducted in cells derived from participants in the Parkinson Progression Markers Initiative (PPMI) study, will generate data that define the underlying molecular disease networks.
Hypothesis: We hypothesize that systems-based approaches, combined with complex genetics, will provide critical information regarding the molecular processes underlying PD.
Study Design: Cells donated by PPMI volunteers will be cultured and grown into neurons similar to those found in the human brain. A molecular characterization of these cells will provide a map of how the various genetic changes linked to PD interact at the cellular and molecular level to cause disease.
Impact on Diagnosis/Treatment of Parkinson’s disease: This work has the potential to inform diagnosis by defining molecular subtypes of disease. By identifying key networks involved in the disease, this work could lead to viable targets for therapeutic intervention.
Next Steps for Development: The clinical application of these results will rely on the testing of potential therapeutics against identified targets. This system can also be used to test whether an intervention is on target and to detect any unanticipated and likely deleterious off-target side effects.