In vivo cell specific gene silencing in the liver using novel siRNA-loaded biodegradable nanohydrogel particles

Background: Efficient in vivo transport of active siRNA to specific liver cells remains challenging. We have designed a new generation of NHP (bio-NHP), this time with biodegradable and ameliorated biocompatibility properties compared to the recent published NHP [Kaps L et al, Adv. Health. care mat.], that show promising in vitro and in vivo performance in one-on-one studies to NHP. Bio-NHP exhibit all NHP's positive characteristics like avoidance of carrier aggregation under physiological conditions and gene silencing in vitro. Results: Bio-NHP composed of block copolymers with acid-labile ketal linker in the i…