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Definition
By Mayo Clinic staffGene therapy involves altering the genes inside your body's cells to stop disease. Genes contain your DNA — the code that controls much of your body's form and function. Your cells use the information from your genes to manufacture proteins that do the work in your body, from making you grow taller to regulating your body systems. Throughout your life, your genes turn on and off as needed to control cell activity.
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 make changes in your body so it's better able to combat disease. Gene therapy holds promise for treating a wide range of diseases, including cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.
Researchers are still learning about how gene therapy works and the best way to administer gene therapy. Currently, in the United States, gene therapy is available only as part of a clinical trial.
