Revisiting Gene Silencing in ALS: From SOD1 Biology to Next‑Generation CNS RNA Platforms
- Re‑examining SOD1 as a therapeutic target in ALS using updated gene‑silencing and delivery approaches informed by recent preclinical work
- Development of a physiological miRNA‑based gene silencing platform using multi‑guide designs to modulate single genes or ALS‑relevant pathways
- Preclinical efficacy and expression data in ALS mouse models, including opportunities for combining gene silencing with protein supplementation
- Engineering a BBB‑crossing capsid to enable durable, pan‑CNS delivery of RNA therapeutics for ALS and related neurodegenerative diseases