Delivering the Goods

Gene Delivery Systems

Stratagene's AdEasy adenoviral vector system One of the most promising approaches to treating genetic diseases, infectious diseases, and cancer is gene therapy, in which genes are transferred directly to an organism. These transferred genes may replace defective genes or target cancerous tissue for selective inhibition of gene activity. Gene therapy has the potential to treat the cause of disease at the genetic level. In ex vivo gene therapy, cells with defective genes are removed from a patient for transfection with wild-type copies of the affected gene. The treated cells are then returned to the patient. In vivo gene therapy involves direct administration of a gene or packaged gene to the patient. The biological agents used for gene therapy are generally cloned DNA sequences but also can include oligonucleotides or ribozymes. The field of gene therapy has shown rapid growth; only 10 years have passed since the first ...