NeurosciencebioRxivPreprint
AAV gene therapy for GBA-PD and Gaucher Disease
Swathi Ayloo, Jae Cheon Ryu, Shih-Ching Chou…·Shyam S. Ramachandran
This paper describes an AAV-mediated gene therapy strategy for GBA1-PD and Gaucher Disease, demonstrating robust GCase replenishment and cross-correction in preclinical models.