Cationic liposome (CL) carriers of nucleic acids are primarily studied because of their applications in gene delivery and gene silencing with CL-DNA and CL-siRNA (short-interfering RNA) complexes, respectively, and their implications to ongoing clinical gene therapy trials worldwide [1-3]. A series of synchrotron-based small-angle-x-ray scattering studies has revealed that CL-nucleic acid complexes spontaneously assemble into distinct novel liquid crystalline phases of matter. We will describe recent fluorescence optical imaging experiments and transfection assays using surface-functionalized CL-DNA nanoparticles interacting with cells. The functionalization, which is achieved through custom synthesis of peptide-PEG-lipids and hydrolysable-PEG-lipids, is intended to address and overcome surface targeting and endosomal escape barriers to gene delivery with PEGylated complexes. Funding provided by NIH.

Surface-functionalized cationic liposome-DNA complexes: Strategies for optimal targetting and endosomal escape for gene delivery

243rd National Meeting (2012)

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