The possibilities of gene therapy hold much promise. Clinical trials of gene therapy in people have shown some success in treating certain diseases, such as:
- 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
- Targeting the correct cells
- Reducing the risk of side effects
Gene therapy continues to be a very important and active area of research aimed at developing new, effective treatments for a variety of diseases.
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.