AAV gene therapy for GBA-PD and Gaucher Disease
Swathi Ayloo, Jae Cheon Ryu, Shih-Ching Chou, Lydia Blatnik, Erik Wischhof, Jie Bu, Lilu Guo, Mahmud Hossain, Dhiman Ghosh, William McCarty, Ann Byrne, Lilly Chai, J T Clarke, Can Kayatekin, Bailin Zhang, , 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.
bioRxiv · Jun 17, 2025 · PreprintNeuroscience