Wen-Xuan Guo

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.