The possibilities of gene therapy hold much promise. To date, however, that promise has not been realized.
Clinical trials of gene therapy in people have shown some success in treating:
- Severe combined immune deficiency
- Blindness caused by retinitis pigmentosa
But, several significant barriers stand in the way of gene therapy becoming a reliable form of treatment, including:
- Finding a reliable way to get genetic material into cells
- Reducing the risk of side effects
- Targeting the correct cells
Gene therapy continues to be a very active area of research.
Jan. 05, 2013
- Gene therapy. Genetics Home Reference. http://ghr.nlm.nih.gov/handbook/therapy. Accessed Nov. 12, 2012.
- Gene therapy for cancer: Questions and answers. National Cancer Institute. http://cancer.gov/cancertopics/factsheet/Therapy/gene. Accessed Nov. 12, 2012.
- Goldman L, et al. Cecil Medicine. 24th ed. Philadelphia, Pa.: Saunders Elsevier; 2012. http://www.mdconsult.com/das/book/body/191371208-2/0/1492/0.html#. Accessed Nov. 12, 2012.
- FAQs. American Society of Gene & Cell Therapy. http://www.asgct.org/general-public/educational-resources/faqs. Accessed Nov. 12, 2012.
- Rivat C, et al. Gene therapy for primary immunodeficiencies. Human Gene therapy. 2012;23:668.
- Garg S. Retinitis pigmentosa: Treatment. http://www.uptodate.com/index. Accessed Dec. 11, 2012.
- Kalos M, et al. T cells with chimeric antigen receptors have potent antitumor effects and can establish memory in patients with advanced leukemia. Science Translational Medicine. 2011;3:1.
- Russell SJ (expert opinion). Mayo Clinic, Rochester, Minn. Dec. 11, 2012.