Pre-clinical evaluation of a gene therapy candidate for SOD1-ALS shows improved survival and signs of inflammation in the CNS of treated mice.
Sonia Pezet, Jennifer Hua, Thibaut Marais, Marine Delamare, Sahar Elouej, Julia P. Lemos, Alexia Castiglione, Stéphanie Astord, Mathilde Cohen-Tannoudji, Georges Arielle Pêche, Mara Rigamonti, Nathalie Danièle, Genries-Ferrand Sandrine, Laurine Buscara, Antonia Ratti, , Maria Grazia Biferi
This study evaluates a gene therapy for SOD1-ALS, showing improved survival and CNS inflammation in treated mice with dose-dependent hSOD1 reduction.
bioRxiv · Oct 16, 2025 · PreprintPharmacology & Toxicology