(SUPPLEMENT) The Foundational Data Initiative

Study Rationale: This project aims to produce foundational data that will provide a resource for understanding the molecular basis of Parkinson’s disease (PD). The work, conducted in cells collected from subjects in the Parkinson Progression Markers Initiative (PPMI) study, will generate data that define the underlying molecular disease networks. This information should point toward targets for therapeutic intervention.

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 subjects from the PPMI will be cultured and grown into nerve cells similar to those found in the human brain. Using a variety of techniques to characterize 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 diagnoses by defining molecular subtypes of PD. Most promisingly, it should identify key networks involved in the disease and to highlight viable targets for therapeutic intervention.

Next Steps for Development: The results will facilitate the testing of novel therapeutics against identified targets. This system can also be used to test whether an intervention reaches its intended target, and whether it precipitates any unanticipated or potentially deleterious off-target side effects.