Cationic liposomes (CLs) are studied worldwide as carriers of DNA and short interfering RNA for gene delivery and gene silencing, and related clinical trials are ongoing. Optimization of transfection efficiency and silencing efficiency requires elucidation of the interactions of CL–nucleic acid nanoparticles (NPs) with cell membranes including events leading to release of active nucleic acids within the cytoplasm. Synchrotron x-ray scattering has revealed the distinct liquid crystalline phases of CL–nucleic acid complexes including the lamellar, inverse hexagonal, hexagonal, and gyroid cubic phases. The talk will describe experiments with surface-functionalized PEGylated CL–DNA nanoparticles (NPs) (Majzoub et al., Biomaterials 2014), including fluorescence microscopy colocalization with members of the Rab GTPases, which has revealed CL–DNA pathways and interactions with cells (Majzoub et al. submitted paper). The functionalization, achieved through custom synthesis, is intended to address and overcome cell targeting and endosomal escape barriers to nucleic acid delivery faced by NPs designed for in vivo applications. Funded by NIH R01 GM 59288.

Functionalized lipid–nucleic acid nanoparticles for delivery applications

249th National Meeting (2015)

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