Restoring Parkin Function: An AAV Gene Therapy Approach for Early-Onset Parkinson's Disease
Sambuddha Basu, Tyler G. Demarest, Nicholas J. Gattone, Alexandra J Gilsrud, Brittany Wicks, Apeksha Khatiwada, Mohammad Nayal, Renee C. Gentzel, Daniel Cohen, Eric Wildon Kostuk, Derek P. Narendra, Pedro Gonzalez‐Alegre, Maria Grazia Biferi, Elizabeth A. Ramsburg
This paper investigates AAV-mediated PRKN gene replacement in Parkin-deficient models to restore mitophagy and demonstrate a feasible gene therapy approach for early-onset Parkinson's disease.
bioRxiv · Jul 13, 2026 · PreprintNeuroscience