History of Gene Therapy

  • Gerhard Bauer
  • Joseph S. Anderson
Part of the SpringerBriefs in Biochemistry and Molecular Biology book series (BRIEFSBIOCHEM)


Gene therapy had its start in the 1970s, including the creation of a regulatory body for gene therapy, the Recombinant DNA Advisory Committee (RAC). The first retroviral-mediated gene therapy clinical trials started in 1990, and the first stem cell gene therapy clinical trial (ADA deficiency), also retrovirally mediated, was initiated in 1994. The first stem cell gene therapy clinical trial for pediatric HIV was conducted in 1997, followed by a stem cell gene therapy for HIV clinical trial in adults with lymphoma using complete marrow ablation in 1999. In October of 1999, the death of Jesse Gelsinger in a clinical trial applying adenoviral vector in vivo was reported. In 2002, leukemia induced by insertional mutagenesis of an integrated retroviral vector in children treated with stem cell gene therapy for X-linked SCID in France was reported.


Gene Therapy Gauche Disease Gene Therapy Study Gene Therapy Protocol Gene Therapy Clinical Trial 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.


  1. 22.
    Fletcher JC (1990) Evolution of ethical debate about human gene therapy. Hum Gene Ther 1(1):55–68CrossRefPubMedGoogle Scholar
  2. 23.
    Friedmann T (2001) Stanfield Rogers: insights into virus vectors and failure of an early gene therapy model. Mol Ther 4(4):285–288CrossRefPubMedGoogle Scholar
  3. 24.
    Jacobs P (1980) Doctor tried gene therapy on two humans. Washington Post, pp A1, A15Google Scholar
  4. 25.
    Protection of human subjects; reports of the President’s Commission for the Study of Ethical Problems in Medicine and Biomedical and Behavioral Research—Office of the Assistant Secretary for Health, HHS. Notice of availability of reports. Fed Regist 48(146):34408–34412 (1983)Google Scholar
  5. 26.
    Walters L (1991) Human gene therapy: ethics and public policy. Hum Gene Ther 2(2):115–122CrossRefPubMedGoogle Scholar
  6. 27.
    Recombinant DNA research; actions under guidelines; notice—E. Points to consider for protocols for the transfer of recombinant DNA into the genome of human subjects. Fed Regist 55(41):7443–7447 (1990)Google Scholar
  7. 28.
    Rosenberg SA et al (1990) Gene transfer into humans—immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med 323(9):570–578CrossRefPubMedGoogle Scholar
  8. 29.
    Culver KW et al (1991) Correction of ADA deficiency in human T lymphocytes using retroviral-mediated gene transfer. Transplant Proc 23(1 Pt 1):170–171PubMedGoogle Scholar
  9. 30.
    Rosenberg SA (1992) Gene therapy of cancer. Important Adv Oncol 17–38Google Scholar
  10. 31.
    Kohn DB et al (1998) T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates. Nat Med 4(7):775–780CrossRefPubMedCentralPubMedGoogle Scholar
  11. 32.
    Malech HL et al (1997) Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease. Proc Natl Acad Sci U S A 94(22):12133–12138CrossRefPubMedCentralPubMedGoogle Scholar
  12. 33.
    Dunbar CE et al (1998) Retroviral transfer of the glucocerebrosidase gene into CD34+ cells from patients with Gaucher disease: in vivo detection of transduced cells without myeloablation. Hum Gene Ther 9(17):2629–2640CrossRefPubMedGoogle Scholar
  13. 34.
    Cornetta K et al (1992) Retroviral-mediated gene transfer of bone marrow cells during autologous bone marrow transplantation for acute leukemia. Hum Gene Ther 3(3):305–318CrossRefPubMedGoogle Scholar
  14. 35.
    Brenner MK et al (1993) Gene marking to determine whether autologous marrow infusion restores long-term haemopoiesis in cancer patients. Lancet 342(8880):1134–1137CrossRefPubMedGoogle Scholar
  15. 36.
    Brenner MK et al (1993) Gene-marking to trace origin of relapse after autologous bone-marrow transplantation. Lancet 341(8837):85–86CrossRefPubMedGoogle Scholar
  16. 37.
    Kohn DB et al (1999) A clinical trial of retroviral-mediated transfer of a rev-responsive element decoy gene into CD34(+) cells from the bone marrow of human immunodeficiency virus-1-infected children. Blood 94(1):368–371PubMedGoogle Scholar
  17. 38.
    Rossi JJ, June CH, Kohn DB (2007) Genetic therapies against HIV. Nat Biotechnol 25(12):1444–1454CrossRefPubMedGoogle Scholar
  18. 39.
    Podsakoff GM et al (2005) Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(+) cells. Mol Ther 12(1):77–86CrossRefPubMedGoogle Scholar
  19. 40.
    Lehrman S (1999) Virus treatment questioned after gene therapy death. Nature 401(6753):517–518CrossRefPubMedGoogle Scholar
  20. 41.
    Nelson D, Weiss R (2000) Penn researchers sued in gene therapy death: teen’s parents also name ethicist as defendant. Washington Post, p A3Google Scholar
  21. 42.
    Cavazzana-Calvo M, Thrasher A, Mavilio F (2004) The future of gene therapy. Nature 427(6977):779–781CrossRefPubMedGoogle Scholar
  22. 43.
    Zaia JA, Federoff HJ (2009) Death in a gene-therapy trial. N Engl J Med 361(18):1811CrossRefPubMedGoogle Scholar

Copyright information

© Gerhard Bauer and Joseph S. Anderson 2014

Authors and Affiliations

  • Gerhard Bauer
    • 1
  • Joseph S. Anderson
    • 1
  1. 1.University of California DavisSacramentoUSA

Personalised recommendations