ABSTRACT

Gene therapy represents a novel approach to treatment using DNA as a drug. It is designed either to alleviate the genetic defect in cells or to provide additional protective effect. Gene therapy is defined as an approach to treating, curing, or preventing disease by changing the expression of a gene. Given that genes regulate all the basic physiological processes in the body, there is tremendous potential for genes to be employed as therapeutic agents.1 The two broad divisions of gene therapy are the somatic and the germ line approaches. In the somatic gene therapy, the transformations are not conserved or passed on to the future generations.2,3 Exogenously administered DNA rarely integrates with the chromosomes of the host genome and persists as an extrachromosomal element (episome) capable of expressing gene products for a period of time before it is eliminated from the host cell by nuclease degradation.3