Programmable RNA editing in Parkinson’s disease therapy

Project Summary

Parkinson’s Disease (PD) is a debilitating neurodegenerative disorder causing severe motor dysfunction and cognitive impairment, affecting >10 million people worldwide. Current treatments are symptomatic, and to date, there are no available therapies proven to cure, halt, or slow disease progression. This team will employ a novel therapeutic technique that harnesses the endogenous adenosine deaminase acting on RNA (ADAR) enzymes for site-directed A-to-G editing - to correct pathogenic mutations causing Parkinson’s disease. They will design and optimize a gene therapy product - ADAR-recruiting guide RNAs - which when delivered to patient-derived cells in vitro or mouse models of PD in vivo can repair pathogenic mutations with high efficacy and specificity, and a promising safety profile.