AAV-mediated SIL1 gene therapy prevents cerebellar degeneration and ataxia in a mouse model of Marinesco-Sjögren syndrome
Chiara Pasini, Giada Lavigna, Anna Grasso, Elena Restelli, Alessandro Corbelli, Monica Salio, Lorena Zentilin, Fabio Fiordaliso, Roberto Chiesa
AAV-mediated SIL1 gene therapy prevents cerebellar degeneration and ataxia in a mouse model of Marinesco-Sjögren syndrome by preserving Purkinje cells and reducing ER stress.
bioRxiv · Sep 24, 2026 · PreprintNeuroscience