ALS Compendium

Title: Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS

Year Published: 2013

Observation: Delivery of AAV9 encoding a shRNA to reduce the synthesis of ALS causing SOD1 mutations extended lifespan

Comment: Extended survival of SOD1 mice from AAV9 mediated reduction in SOD1 even when initiated mid disease

Citation: Foust, K. D., Salazar, D. L., Likhite, S., Ferraiuolo, L., Ditsworth, D., Ilieva, H., … Kaspar, B. K. (2013). Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS, 21(12), 2148–2159. doi:10.1038/mt.2013.211

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