Oct 2026
The accessory makes the drug: ASO-delivered siRNA enters the human CNS for ALS.
In a recent article in Nature Medicine, Chen et al. describe a first-in-human study using an ASO tag to deliver a SOD1 siRNA to the central nervous system (CNS), potentially achieving faster, more extensive, and better-tolerated SOD1-lowering in ALS patients.1.
Wen-Xuan Guo, J. Ichida
· Cell Stem Cell · 0 citations