The protease inhibitors used for the treatment of AIDS were also developed through recombinant technology, and similar approaches are in development for the treatment of hepatitis. Looking into the future, Professor White spoke about what this remarkable technology might bring us in the next decade, focussing on gene therapy and inhibitors. Literally, gene therapy is using genes and their products to treat disorders. This could involve replacement of an abnormal gene, as in hemophilia, or the introduction of a new gene that might, for instance, stimulate the immune system to attack cancer cells. The challenge is transferring the new genes into the right cells in a way that they will function properly and not disrupt other genes in the cell.
Most current methods result in the genes being introduced into essentially all tissues of the body.
However, it is possible that the protein made by the new gene could interfere with the functioning of some tissues where that protein is not normally made. Even in the correct tissue the new gene can disrupt proper cell functioning if it inserts itself onto the cell's chromosomes in a location that interferes with another gene. Thus the usefulness of gene therapy is highly dependent on the development of safe and efficient methods for accomplishing gene transfer. This is the main focus of current research in gene therapy. A number of target tissues have been proposed for hemophilia gene therapy.