DNA: Gene therapy


Gene therapy is a recombinant DNA process in which cells are taken from the patient, altered by adding genes, and replaced in the patient, where the genes provide the genetic codes for proteins the patient is lacking.

In the early 1990s, gene therapy was used to correct a deficiency of the enzyme Adenosine Deaminase (ADA). Blood cells called lymphocytes were removed from the bone marrow of two children; then genes for ADA production were inserted into the cells using viruses as vectors. Finally, the cells were reinfused to the bodies of the two children. Once established in the bodies, the gene-altered cells began synthesizing the enzyme ADA and alleviated the deficiency.

Gene therapy has also been performed with patients with melanoma (a virulent skin cancer). In this case, lymphocytes that normally attack tumors are isolated in the patients and treated with genes for an anticancer protein called tumor necrosis factor.The genealtered lymphocytes are then reinfused to the patients, where they produce the new protein which helps destroy cancer cells. Approximately 2000 single-gene defects are believed to exist, and patients with these defects may be candidates for gene therapy.