ABSTRACT
INTRODUCTION The full potential of gene therapy in medicine is being realized today. It is defined as the use of genes or genetic material (DNA, RNA, oligonucleotides) to treat a disease state, generally a genetic-based disease (1-3). Genes are introduced into cells or tissues either to inhibit undesirable gene expression or to express therapeutic proteins (3). Target disease states for gene delivery can be broadly categorized into two major classes: inherited and acquired. Inherited disease states are limited to sickle cell anemia, hemophilia, cystic fibrosis, Huntington’s disease, and errors of metabolism; acquired diseases include cancer, HIV infection, and diabetes. The two standard procedures used in gene delivery are addition/replacement and ablation (4). While performing the former, a normal gene is introduced into the cell type to replace activity of the defective gene (4). On the contrary, ablation deals with destroying undesired cells, as in cancer.
