NeurosciencebioRxivPreprint
AAV-mediated SIL1 gene therapy prevents cerebellar degeneration and ataxia in a mouse model of Marinesco-Sjögren syndrome
Chiara Pasini, Giada Lavigna, Anna Grasso…·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.
DOI 10.64898/2026.09.14.751587