Preemptive SOD1 Silencing via Neonatal Intramuscular AAV Therapy Modifies Disease Trajectory in an ALS Mouse Model
Xueqi Gong, Yicheng Xie, Wenyuan Wang, Tao Xu
This study uses intramuscular AAV therapy to silence SOD1 in neonatal mice, delaying ALS onset and improving survival and motor function.
bioRxiv · Oct 17, 2025 · PreprintNeuroscience