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Risks
By Mayo Clinic staffGene therapy poses a number of risks. First, the way the genes are delivered can be problematic. A gene can't easily be inserted directly into your cells. Rather, it usually has to be delivered using a carrier, called a vector. The most common gene therapy vectors are viruses because they can recognize certain cells and carry genetic material into the cells' genes. Researchers are trying to take advantage of this unique capability by removing the original disease-causing genes from the viruses, replacing them with the genes needed to stop disease, and then inserting the altered viruses into a person's diseased cells, where they can deliver their genetic material.
This technique presents the following risks:
- Immune response. Your body's immune system may see the newly introduced viruses as intruders and attack them. This may cause inflammation, toxicity and, in severe cases, organ failure.
- Viral spread. Because viruses can affect more than one type of cells, it's possible that the viral vectors may infect cells in addition to the ones for which they were intended — just those containing mutated or missing genes. If this happens, healthy cells may be damaged, causing other illness or diseases, including cancer.
- Reversion of the virus to its original form. It's possible that once introduced into the body, the viruses may recover their original ability to cause disease.
- Possibility of inducing a tumor. If the new genes get inserted in the wrong spot in the genome, there is a chance that the insertion might lead to tumor formation. This has occurred occasionally in some clinical trials.
Another risk of gene therapy is that the new DNA introduced into your body may affect your reproductive cells — egg cells in women, sperm cells in men. This could result in genetic changes that could affect children you have after treatment.
The gene therapy clinical trials under way in the U.S. are closely monitored by the Food and Drug Administration and the National Institutes of Health to ensure the safety of those who participate in the studies.
- 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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