Parkinson's disease: Genetic-driven therapeutic approaches

Abstract Disease modification remains a major unmet need in Parkinson's disease (PD) therapeutics. Two decades ago, the discovery of familial mutations responsible for PD radically changed the field and provided a foundation to uncover the dysfunctional molecular pathways amenable to therapeutic intervention. This chapter highlights how genetics informs understanding of pathogenic mechanisms in Parkinson's and leads to the development of therapeutics aimed at halting or slowing the underlying disease process. We describe three genetic discoveries (SNCA, GBA, and LRRK2) that have led to novel therapeutics currently entering the clinic.