Molecular BiologyMolecular Therapy — Nucleic AcidsPeer-reviewed
Delivery of GalNAc-conjugated splice-switching ASOs to non-hepatic cells through ectopic expression of asialoglycoprotein receptor
Juergen Scharner, Sabrina Qi, Frank Rigo…·Adrian R. Krainer
This study demonstrates that GalNAc-conjugated ASOs can target cancer cells expressing ectopic ASGP-R, improving splice-switching ASO potency in vitro and in tumor models.
29 citations