What you can expect

Currently, the only way for you to receive gene therapy is to participate in a clinical trial. Clinical trials are research studies that help doctors determine whether a gene therapy approach is safe for people. They also help doctors understand the effects of gene therapy on the body.

Your specific procedure will depend on the disease you have and the type of gene therapy being used.

For example, in one type of gene therapy:

  • You may have blood drawn or you may need bone marrow removed from your hipbone with a large needle.
  • Then, in a lab, cells from the blood or bone marrow are exposed to a virus or another type of vector that contains the desired genetic material.
  • Once the vector has entered the cells in the lab, those cells are injected back into your body into a vein or into tissue, where your cells take up the vector along with the altered genes.

Viruses aren't the only vectors that can be used to carry altered genes into your body's cells. Other vectors being studied in clinical trials include:

  • Stem cells. Stem cells are the cells from which all other cells in your body are created. For gene therapy, stem cells can be trained in a lab to become cells that can help fight disease.
  • Liposomes. These fatty particles have the ability to carry the new, therapeutic genes to the target cells and pass the genes into your cells' DNA.
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.