Targeted siRNA-Lactobionic Lipid Nanoparticles with Novel Ionizable Lipid Inhibit Antithrombin III Expression in Hepatocytes
This study demonstrates that a novel targeted lipid nanoparticle formulation, incorporating a morpholine-based ionizable lipid and a lactobionic-octadecylamine conjugate, efficiently delivers siRNA to hepatocytes to achieve robust knockdown of antithrombin III with low cytotoxicity, offering a promising therapeutic strategy for bleeding disorders.