Gene therapy involves altering the genes inside your body's
cells in an effort to treat or stop disease. Genes contain your DNA — the code that controls much of your body's form and function, from making you grow taller to regulating your body systems. Genes that don't work properly can cause disease. Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve your body's ability to fight disease. Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS. Researchers are still studying how and when to use gene therapy. Currently, in the United States,
gene therapy is available only as part of a clinical trial. Gene therapy is used to correct defective genes in order to cure a disease or help your body better fight disease. Researchers are investigating several ways to do this, including: Replacing mutated genes. Some
cells become diseased because certain genes work incorrectly or no longer work at all. Replacing the defective genes may help treat certain diseases. For instance, a gene called p53 normally prevents
tumor growth. Several types of
cancer have been linked to problems with the p53 gene. If doctors could replace the defective p53 gene that might trigger the
cancer cells to die. Fixing mutated genes. Mutated genes that cause disease could be turned off so that they no longer promote disease, or healthy genes that help prevent disease could be turned on so that they could inhibit the disease. Making diseased
cells more evident to the immune system. In some cases, your
immune system doesn't attack diseased
cells because it doesn't recognize them as intruders.
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