Our goal is to determine if a clinically safe FTI called lonafarnib can enhance cellular degradation and eliminate toxic a-synuclein in PD-patient derived midbrain cultures and mouse models. We will establish the optimal dose of lonafarnib that is required to activate degradation pathways in the brain. Simultaneously, we will establish blood biomarkers to track target engagement of lonafarnib in mice, which may provide a proxy for brain activity. Our studies may guide future clinical trials by providing sensitive biochemical assays that can be used in conjunction with clinical measures to determine drug efficacy
|Effective start/end date||7/1/21 → 12/31/22|
- Cure Parkinson's Trust (Agmt 10/20/2021)
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