Liposomal carriers gene therapy

In this chapter, we provide an overview of our recent efforts to develop a fundamental science base for the design and preparation of optimal lipid-based carriers of DNA and siRNA for gene therapy and gene silencing. We employ synthesis of custom multivalent lipids, synchrotron X-ray diffraction (XRD) techniques, optical and cryo-electron microscopy, as well as biological assays in order to correlate the structures, chemical, and biophysical properties of cationic liposome (CL)-NA complexes to their biological activity and to clarify the interactions between CL-NA complexes and cellular components. Earlier work has been reviewed elsewhere [1-7] and will not be covered exhaustively here. 

Fundamentally, gene delivery systems can be differentiated into viral carriers (adenovirus, lentivirus, etc.) and non-viral vectors (complexes, micelles, liposomes, nanoparticles, etc.) [8]. Following are the major requirements for both polymers and nanoparticulate systems intended for gene therapy. 

Liposomes, phospholipid nanosized bubbles with a bilayered membrane structure, have drawn a lot of interest as pharmaceutical carriers for drugs and genes. Current liposomology includes multiple areas, from clinical application of the liposomal drugs to the development of various multifunctional liposomal systems to be used in therapy and diagnostics. This chapter briefly discusses the pharmaceutical application of liposomes and provides an overview of various liposomal products currently under development at experimental and preclinical level. 

---