AAV delivery of GBA1 suppresses alpha-synuclein accumulation in Parkinson's disease models and restores motor dysfunction in a Gaucher's disease model
Takuro Okai, Sho Sato, Mugdha Deshpande, Shin-ichi Matsumoto, Miyu Nakayama, Syunsuke Yamamoto, Bettina Strack-Logue, Takeshi Hioki, Maiko Tanaka, Gabriele Proetzel
This paper shows that delivering GBA1 via AAV reduces alpha-synuclein aggregates and restores motor function in disease models.
bioRxiv · Jun 25, 2024 · PreprintNeuroscience