Overview

Gene therapy involves altering the genes inside your body's cells in an effort to treat or stop disease.

Genes contain your DNA — the code that controls much of your body's form and function, from making you grow taller to regulating your body systems. Genes that don't work properly can cause disease.

Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve your body's ability to fight disease. Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.

Researchers are still studying how and when to use gene therapy. Currently, in the United States, gene therapy is available only as part of a clinical trial.

Sept. 13, 2016
References
  1. Gene therapy. Genetics Home Reference. http://ghr.nlm.nih.gov/handbook/therapy. Accessed July 21, 2016.
  2. What is gene therapy? National Cancer Institute. http://cancer.gov/ about-cancer/treatment/types/immunotherapy/bio-therapies-fact-sheet#q8. Accessed July 22, 2016.
  3. Chuah M, et al. Recent progress in gene therapy for hemophilia. Human Gene Therapy. 2012;23;557.
  4. FAQs. American Society of Gene & Cell Therapy. http://www.asgct.org/general-public/educational-resources/faqs. Accessed July 22, 2016.
  5. Cicalese M, et al. Clinical applications of gene therapy for primary immunodeficiencies. Human Gene Therapy. 2015;26:210.
  6. 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.
  7. 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.
  8. Russell SJ (expert opinion). Mayo Clinic, Rochester, Minn. Aug. 29, 2016.