A PEG-Based Biocompatible Block Catiomer with High Buffering Capacity for the Construction of Polyplex Micelles Showing Efficient Gene Transfer toward Primary Cells

摘要

Nonviral gene vectors from synthetic catiomers (polyplexes) are a promising alternative to viral vectors.In particular,many recent efforts have been devoted to the construction of biocompatible polyplexes for in vivo nonviral gene therapy.A promising approach in this regard is the use of polyethylene glycol) (PEG)-based block catiomers,which form a nanoscaled core-shell poly-plex with biocompatible PEC palisades.In this study,a series of PEC-based block catiomers with different amine functionalities were newly prepared by a simple and affordable synthetic procedure based on an aminolysis reaction,and their utility as gene carriers was investigated.This study revealed that the block catiomers carrying the ethylenediamine unit at the side chain are capable of efficient and less toxic transfection even toward primary cells,highlighting critical structural factors of the cationic units in the construction of polyplex-type gene vectors.Moreover,the availability of the polyplex micelle for transfection with primary osteoblasts will facilitate its use for bone regeneration in vivo mediated by nonviral gene transfection.