Gene therapy

Gene therapy is a treatment that 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, 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, including 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.


Why it's done Risks What you can expect Results

Gene therapy is used to correct defective genes in order to cure a disease or to 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 in your body. 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 can 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. Doctors could use gene therapy to train your immune system to recognize the cells that are a threat.
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