Gene therapy

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Results

By Mayo Clinic staff

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
  • Hemophilia
  • Blindness caused by retinitis pigmentosa
  • Leukemia

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.

What you can expect

  • See Also

    • Hand Scheduled
    Related Links
    Section Focus
  • Hemophilia

    • Related Guides
References
  1. Gene therapy. Genetics Home Reference. http://ghr.nlm.nih.gov/handbook/therapy. Accessed Nov. 12, 2012.
  2. Gene therapy for cancer: Questions and answers. National Cancer Institute. http://cancer.gov/cancertopics/factsheet/Therapy/gene. Accessed Nov. 12, 2012.
  3. 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.
  4. FAQs. American Society of Gene & Cell Therapy. http://www.asgct.org/general-public/educational-resources/faqs. Accessed Nov. 12, 2012.
  5. Rivat C, et al. Gene therapy for primary immunodeficiencies. Human Gene therapy. 2012;23:668.
  6. Garg S. Retinitis pigmentosa: Treatment. http://www.uptodate.com/index. Accessed Dec. 11, 2012.
  7. 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.
  8. Russell SJ (expert opinion). Mayo Clinic, Rochester, Minn. Dec. 11, 2012.
MY00105 Jan. 5, 2013

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