Gene Supplement and Silencing in FTD and ALS: From Basic Biology to Next Generation CNS RNA Platforms
- Choice of PGRN and SOD1 as therapeutic targets using precision-guided gene supplement and silencing and adeno-associated viral delivery
- Engineering a physiological miRNA based gene silencing platform using multi guide designs to modulate single genes or multiple genes in ALS relevant pathways
- Preclinical efficacy and expression data in ALS mouse models, including opportunities for combining gene silencing with protein supplementation
- Advances in BBB crossing capsids enable durable, pan CNS delivery of RNA therapeutics for ALS and related neurodegenerative diseases