Local administration of a novel siRNA modality into the CNS extends survival and improves motor function in the SOD1G93A mouse model for ALS
C. Duan, M. Kang, X. Pan, Z. Gan, V. Huang, G. Li, R. F. Place, L.-C. Li
This study demonstrates that a novel siRNA modality, conjugated with an accessory oligonucleotide, improves survival and motor function in a mouse model of ALS when delivered locally to the CNS.
bioRxiv · Feb 27, 2023 · PreprintCell Biology