A triple chain polycationic peptide-mimicking amphiphile - efficient DNA-transfer without co-lipids.

摘要: Non-viral gene delivery in its current form is largely dependent upon the ability of a vehicle to protect cargo extracellular environment and release it efficiently inside target cell. Also simple system required simplify GMP conform production if marketing authorization striven for. This work addresses these problems. We have developed synthetic polycationic peptide-mimicking amphiphile, namely DiTT4, which shows efficient transfection rates good biocompatibility without use co-lipid formulation. The lipid–nucleic acid complex (lipoplex) was characterized at structural (electron microscopy), physical (laser Doppler velocimetry atomic force microscopy) molecular levels (X-ray scattering). Stability studies lipoplexes presence serum heparin indicated stable formation capable protecting against milieu. Hemocompatibility (hemolysis, complement activation erythrocyte aggregation) demonstrated formulation for systemic administration. efficiency assessed vitro using GFP assay confocal laser scanning microscopy studies. With chorioallantoic membrane model, an animal replacement model according 3R strategy (replacement, refinement, reduction), initial vivo experiments were performed demonstrate fast tissues excellent biocompatibility.