ABSTRACT

Gene therapy is a novel form of molecular medicine that will have a major impact on human health. The minimum requirement for gene therapy is sustained production of the therapeutic gene product without any harmful side effects. One alternative vector for stable gene expression is the adeno-associated virus vector. There are a number of somatic tissues that have been used to transfer genes, but the precise choice of the tissue will be dictated by the nature of the disease. The transduced somatic tissue can be implanted where only local delivery of the therapeutic protein is required. Although a wide variety of somatic tissues has been identified for delivering genes, the major hurdle is still the mode of delivery of the therapeutic genes. Gene therapy approach involves DNA transfection with calcium phosphate, DEAE dextrana and cationic lipids, direct DNA injection with or without the aid of cationic lipids, electroporation, ballistic guns delivering gold-coated DNA particles, microinjection, liposomes, and receptor-mediated gene transfer.