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Why it's done
By Mayo Clinic staffGene 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 missing or mutated genes. This is currently the most common gene therapy approach. Some cells become diseased because certain genes have been permanently turned off. Other cells may be missing certain genes. Researchers hope that replacing missing or defective genes can help treat certain diseases. For instance, a common tumor suppressor gene called p53 normally prevents tumor growth in your body. Several types of cancer have been linked to a missing or inactive p53 gene. If doctors could replace p53 where it's missing, that might trigger the cancer cells to die.
- Changing the regulation of a gene. 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. Using gene therapy, doctors could potentially infuse mutated cells with genes that make them more recognizable to your immune system. Or enhancements could be made to immune cells to make it easier for them to recognize mutated cells that are a threat.
References
- Gene therapy. Centre for Genetics Education. http://www.genetics.edu.au/pdf/factsheets/fs27.pdf. Accessed Sept. 7, 2010.
- Gene therapy for cancer: Questions and answers. National Cancer Institute. http://www.cancer.gov/cancertopics/factsheet/Therapy/gene. Accessed Sept. 7, 2010.
- Gene therapy. Human Genome Project Information. http://www.ornl.gov/sci/techresources/Human_Genome/medicine/genetherapy.shtml. Accessed Sept. 7, 2010.
- Genetics home reference. National Library of Medicine. http://ghr.nlm.nih.gov/handbook. Accessed Sept. 7, 2010.
- Wieben ED (expert opinion). Mayo Clinic, Rochester, Minn. Sept. 18, 2010
- About clinical trials. American Society of Gene & Cell Therapy. http://www.asgct.org/educational_resources/clinical_trials.php. Accessed Sept. 24, 2010.
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