Cell-resolved pharmacodynamic profiling reveals tissue- and cell-population-specific activity of antisense oligonucleotide chemical conjugates in vivo
Shuzhi Cheng, Jennifer Frommer, Ayad Eddaoudi, Panayiota Constandinou, Matthew Wood, Francesco Muntoni, Haiyan Zhou
This study evaluates cell-specific activity of antisense oligonucleotide conjugates in vivo using a reporter mouse model and finds that palmitate conjugation shows broad extrahepatic activity and preferential skeletal muscle targeting.
bioRxiv · Nov 2, 2026 · PreprintMolecular Biology