Open access
Aug 2026
ESCRT-I complex enhances therapeutic efficacy of ASO drugs by regulating endosomal escape and glucose homeostasis
Antisense oligonucleotide (ASO) therapeutics silence gene expression through RNase H-mediated mRNA degradation or steric blockade, yet their clinical efficacy is limited by inefficient intracellular trafficking, with <1% of internalized ASOs escaping endosomes to access target RNA. A deeper understanding of the cellula...
Le Tra Giang Nguyen, Sherouk M. Tawfik, Jing Jin et al.
· Molecular Therapy: Nucleic A... · 0 citations