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
  • 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
  • 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
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  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. 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.
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