Gene Therapy - The use of DNA as a Drug

Abstract

GENE therapy represents a fundamentally new way to treat a disease. The use of genetic material (genes), which can express a protein in the cell or interfere with the synthesis of a protein in the cell, in order to treat a disease, is gene therapy. Replacing a defective gene with a normal gene and thus restoring the lost gene function in the patient's body is the essence of gene therapy. Originally conceived as an approach to treat autosomal recessive Mendelian disorders, it is now being applied to a broad range of acquired conditions such as cancers, infections and degenerative disorders. With the understanding of the genetic basis of cancer, an entirely new approach to the treatment of cancer using gene transfer techniques has evolved. DNA is now slated to be used as a therapeutic agent to replace defective genes in patients suffering from genetic disorders or to kill tumor cells in cancer patients.