Pharmacology & ToxicologybioRxivPreprint
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…·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.
DOI 10.1101/2025.10.16.682744