Intravenous administration of an engineered AAV9-gene-silencing vector suppresses human SOD1 and extends survival in an ALS mouse model. - Wan F; He J; Ma H; PiresFerreira D; Kumanan V; Lee JS; Chen X; He R; Su Q; Gallagher TL; Zhu S; Cabrera GT; et al. (Nature communications, 2026)