Precision Medicine in Parkinson's Disease

The continually accumulating knowledge of Parkinson's disease (PD) genetics presents potential disease modification opportunities through targeting specific genes and associated metabolic pathways. Glucosylceramidase beta 1-associated PD and leucine-rich repeat kinase 2-associated PD are attractive drug targets, since their respective mutations significantly increase PD risk and, at the same time, are relatively prevalent in the PD population. Here, we review clinical trials and preclinical efforts whose mechanisms target genetic forms of PD, focusing on these 2 genes and their metabolic pathways. Such therapies could also potentially modify sporadic (ie, without a clear genetic risk factor) PD.