The accessory makes the drug: ASO-delivered siRNA enters the human CNS for ALS.

ASO-tagged siRNA successfully delivers SOD1 gene silencing to the human CNS, achieving improved targeting in ALS patients with enhanced tolerability.

• Why it matters: Effective gene silencing in the CNS is critical for treating neurodegenerative diseases like ALS, but delivery methods often face limitations in speed, extent, and safety.
• What they did: Chen et al. conducted a first-in-human study using an antisense oligonucleotide (ASO) tag to deliver siRNA targeting SOD1, a gene implicated in ALS, to the central nervous system.
• The result: The approach enabled faster and more extensive SOD1 reduction with better tolerability, opening new avenues for gene therapy in neurodegenerative disorders.