Thanks to gene therapy research, treatment in the form of enzyme replacement is within months of being on the market, Schaefer said. If it works, the treatment could relieve many of the disease symptoms and stop its progression. Gene therapy, as defined by the American Society of Gene Therapy, is the treatment of disease by either replacing damaged or abnormal genes with normal ones or providing new genetic instructions to help fight disease. As an extension of conventional medicine, the goal of gene therapy is to treat disease by administering genetic material rather than a drug. Theoretically, a disease may be cured by a single administration of gene therapy.
Reality tells a different story.
For more than 10 years, scientists have been working to find gene therapies for such conditions as cancer, arthritis, vascular disease and genetic disorders. As yet, no one has discovered a bankable method. But that hasn't deterred them. The University of Nebraska Medical Center in Omaha has not done clinical gene therapy trials, but scientists there are engaged in some basic research. He is studying how gene therapy can be used to kill breast cancer cells.