| 초록 |
The development of efficient and safe gene delivery vector is a major hurdle in gene therapy. Core-shell nanoparticles with condensed nucleic acid core and cationic acid-degradable polyketal shell was developed and explored for enhanced, synergistic and versatile nucleic acid delivery. Core-shell nanoparticle formulations of polyamine/pDNA polyplexes, dendrimer/pDNA dendriplexes and adeno-associated viruses (AAVs) shelled with cationic acid-degradable polyketal shell achieved enhanced gene delivery efficiencies compared to the cores alone facilitated by efficient cellular uptake, cytosolic release and nuclear trafficking. Moreover, synergistic gene therapy was achieved by co-deliverying cDNA and siRNA. Leukemia cells were efficiently eradicated by simultaneous delivery of cDNA and siRNA both in vitro and in vivo. Lastly, polyketal shell provided versatility to prepare various shell compositions of desired properties. |
