Gene therapy has some potential risks. 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 remove the original disease-causing genes from the viruses, replacing them with the genes needed to stop disease.
This technique presents the following risks:
- Unwanted immune system reaction. Your body's immune system may see the newly introduced viruses as intruders and attack them. This may cause inflammation and, in severe cases, organ failure.
- Targeting the wrong cells. Because viruses can affect more than one type of cells, it's possible that the altered viruses may infect additional cells — not just the targeted cells containing mutated genes. If this happens, healthy cells may be damaged, causing other illness or diseases, such as cancer.
- Infection caused by the virus. It's possible that once introduced into the body, the viruses may recover their original ability to cause disease.
- Possibility of causing a tumor. If the new genes get inserted in the wrong spot in your DNA, there is a chance that the insertion might lead to tumor formation.
The gene therapy clinical trials underway in the U.S. are closely monitored by the Food and Drug Administration and the National Institutes of Health to ensure that patient safety issues are a top priority during research.
Sept. 13, 2016
- Gene therapy. Genetics Home Reference. http://ghr.nlm.nih.gov/handbook/therapy. Accessed July 21, 2016.
- What is gene therapy? National Cancer Institute. http://cancer.gov/ about-cancer/treatment/types/immunotherapy/bio-therapies-fact-sheet#q8. Accessed July 22, 2016.
- Chuah M, et al. Recent progress in gene therapy for hemophilia. Human Gene Therapy. 2012;23;557.
- FAQs. American Society of Gene & Cell Therapy. http://www.asgct.org/general-public/educational-resources/faqs. Accessed July 22, 2016.
- Cicalese M, et al. Clinical applications of gene therapy for primary immunodeficiencies. Human Gene Therapy. 2015;26:210.
- Ghazi N, et al. Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: Results of a phase I trial. Human Genetics. 2016;135;327.
- Schubert M, et al. Chimeric antigen receptor T cell therapy targeting CD19 positive leukemia and lymphoma in the context of stem cell transplantation. Human Gene Therapy. In press. Accessed Aug. 13, 2016.
- Russell SJ (expert opinion). Mayo Clinic, Rochester, Minn. Aug. 29, 2016.