Engineered AAV9 vector targets SOD1 mutations in ALS, extending mouse survival. Gene silencing through a single IV dose shows promise for durable ALS treatment. PMID:42350385, Nat Commun 2026, @NatureComms https://doi.org/10.1038/s41467-026-74169-8 #Medsky #Pharmsky #RNA #ASHG #ESHG 🧪
