Issue 11, 2014

Cationic liposome–nucleic acid complexes for gene delivery and gene silencing

Abstract

Cationic liposomes (CLs) are studied worldwide as carriers of DNA and short interfering RNA (siRNA) for gene delivery and gene silencing, and related clinical trials are ongoing. Optimization of transfection efficiency and silencing efficiency by cationic liposome carriers requires a comprehensive understanding of the structures of CL–nucleic acid complexes and the nature of their interactions with cell membranes as well as events leading to release of active nucleic acids within the cytoplasm. Synchrotron X-ray scattering has revealed that CL–nucleic acid complexes spontaneously assemble into distinct liquid crystalline phases including the lamellar, inverse hexagonal, hexagonal, and gyroid cubic phases, and fluorescence microscopy has revealed CL–DNA pathways and interactions with cells. The combining of custom synthesis with characterization techniques and gene expression and silencing assays has begun to unveil structure–function relations in vitro. As a recent example, this review will briefly describe experiments with surface-functionalized PEGylated CL–DNA nanoparticles. The functionalization, which is achieved through custom synthesis, is intended to address and overcome cell targeting and endosomal escape barriers to nucleic acid delivery faced by PEGylated nanoparticles designed for in vivo applications.

Graphical abstract: Cationic liposome–nucleic acid complexes for gene delivery and gene silencing

Article information

Article type
Perspective
Submitted
06 Aug 2014
Accepted
12 Sep 2014
First published
18 Sep 2014

New J. Chem., 2014,38, 5164-5172

Cationic liposome–nucleic acid complexes for gene delivery and gene silencing

C. R. Safinya, K. K. Ewert, R. N. Majzoub and C. Leal, New J. Chem., 2014, 38, 5164 DOI: 10.1039/C4NJ01314J

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