Polymeric nanoparticles assisted delivery of nucleic acid for targeted gene therapy

Gene therapy refers to the introduction of a therapeutic gene or nucleic acid to modify and regulate the expression of a target gene. This is emerging as a promising and alternative strategy against drug treatment, surgical... [ view full abstract ]

Gene therapy refers to the introduction of a therapeutic gene or nucleic acid to modify and regulate the expression of a target gene. This is emerging as a promising and alternative strategy against drug treatment, surgical approach and enzyme/protein therapy for the treatment of various deadly diseases. According to a fact sheet of World Health Organization (WHO), cardiovascular diseases are causing the highest percent of death rate all over the world. Despite of implementation of many diagnostic techniques and remedies, the success rate against diseases is very low. Gene therapy is come up as a better way to surmount this problem. Further, the therapeutic potential of a gene relies upon its safe and targeted site delivery. For this, several delivery systems have been explored, however limited success have achieved. In current study, we have designed chitosan nanoparticles, a bio-compatible and biodegradable polymer based delivery vectors for siRNA delivery to target cardiovascular diseases. Chitosan would be modified with suitable ligands for targeted delivery of siRNA to mammalian cells, HepG2. The modification would be evaluated by Fourier transform infrared spectroscopy (FT-IR) and nuclear magnetic resonance (NMR). Nanoparticles thus prepared would be characterized for size, shape, surface morphology by DLS, SEM and TEM, respectively. Entrapment efficiency and in vitro gene expression was evaluated by fluorescence microscopy. Further, quantitative analysis of gene expression would be analyzed and this study could be used for the development of an efficient delivery system.