Abstract

LIPOPLEXES: A NON-VIRAL GENE DELIVERY VEHICLE

*Gupta Ravikant

ABSTRACT

A lipoplex (also known as Genosome) is a lipid and DNA complex that is used to deliver genes. It can be a form of non-viral gene therapy as the complex does not require any components of a virus in order to transport genetic material. As a promising strategy for the treatment of many inherited and acquired diseases, gene therapy which is defined as the genetic modification of cells for therapeutic benefit. The aim for gene therapy is to deliver healthy exogenous drugs such as plasmid DNA and single-strand oligonucleotides to replace a missing gene that otherwise have a normal makeup so as to cure genetic diseases, for instance, cystic fibrosis, malignant melanoma, and gaucher's disease. The formation of lipoplexes was a result of the electrostatic interaction between cationic charge from lipids and anionic charges from DNA. It include both large scale production and small scale production. Mechanism of action of lipoplexes include Endocytosis of lipoplexes and then release of lipoplex from endosomes. Various characterization techniques are used for evaluation of lipoplexes like light scattering, electrophoretic mobility, microscopy, diffraction calorimetry, FT-IR, NMR, ESR, FRET and separate characterization parameters for lipoplexes and physicochemical properties and toxicity studies also done. Results shows that good bioavailability, biocompatibility, inhence permeability and targeting achieved. Future of this type of drug deliver system will explore the utilization of nanogenobiotics.

Keywords: gaucher's disease, nanogenobiotics, FT-IR, NMR, ESR, FRET.