Precision Medicine and Genotype-Targeted Therapies in Parkinson’s Disease

Abstract Purpose of Review This article reviews current prospects for disease-modifying therapy in Parkinson’s Disease, with a focus on genotype-targeted treatments for the condition. Recent Findings Over 100 genes and loci are implicated in the pathogenesis of PD. They have varied cellular functions and studies of these roles have yielded insights into the mechanisms underlying PD pathogenesis, with this in turn leading to the identification of multiple possible targets for disease modification. Some of the most promising developments so far have been with GBA1, LRRK2, PINK1/PRKN and SNCA, and therapeutic strategies employing molecular chaperones, antisense oligonucleotides, small molecule inhibitors/activators, and gene therapies among others have been proposed. Summary There remain no currently licensed medications to slow or prevent the progression of Parkinson’s Disease, the second most common neurodegenerative disease worldwide. However, multiple potential genotype-targeted therapeutic options have been developed and have progressed through pre-clinical and clinical phase work, with many trials expected to report shortly.