Immunodeficiency Syndromes: Gene Therapy

Alain Fischer, Department of Pediatric Immunology–Hematology,INSERM 429, Paris, France

Published online: January 2006

DOI: 10.1038/npg.els.0005752

Gene therapy can potentially offer a mean, to correct the immunodeficiences provoked by a number of inherited monogenic diseases. There have recently been successful clinical trials of such treatment.

Keywords: gene therapy; SCCD; immunodeficiencies; T lymphocytes; hematopoietic stem cells; genetic disease

Figure 1. Schematic representation of hematopoiesis.
close
 References
    Blaese RM, Culver KW, Miller AD, et al. (1995) T-lymphocyte-directed gene therapy for ADA–SCID: initial trial results after 4 years. Science 270: 475–480.
    Bordignon C, Notarangelo LD, Nobili N, et al. (1995) Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients. Science 270: 470–475.
    Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, et al. (2000) Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288: 669–672.
    Fischer A (2001) Primary immunodeficiency diseases: an experimental model for molecular medicine. Lancet 357: 1863–1869.
    Kohn DB, Hershfield MS, Carbonaro D, 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. Nature Medicine 4: 775–780.
    Malech HL (2000) Use of serum free medium with fibronectin fragment entranced transduction in a system of gas permeable plastics containers to achieve high levels of retrovirus transduction at clinical scale. Stem Cells 18: 150–156.
 Further Reading
    Candotti F (2000) The potential for therapy of immune disorders with gene therapy. Pediatric Clinics of North America 47: 1389–1407.
    Fischer A (2000) Cautious advances: gene therapy is more complex than anticipated. EMBO Reports 1(4): 294–296.
    Halene S and Kohn DB (2000) Gene therapy using hematopoietic stem cells: Sisyphus approaches the crest. Human Gene Therapy 11: 1259–1267.
    May C, Rivella S, Callegari J, et al. (2000) Therapeutic haemoglobin synthesis in -thalassemic mice expressing lentivirus-encoded human -globin. Nature 406: 82–86.
    Sadelain M, Frassoni F and Riviere I (2000) Issues in the manufacture and transplantation of genetically modified hematopoietic stem cells. Current Opinion in Hematology 7: 364–377.
 Web Links
    ePath Adenosine deaminase (ADA); Locus ID: 100. LocusLink: http://www.ncbi.nlm.nih.gov/LocusLink/LocRpt.cgi?l=100
    ePath Janus kinase 3 (a protein tyrosine kinase, leukocyte) (JAK3); Locus ID: 3718. LocusLink: http://www.ncbi.nlm.nih.gov/LocusLink/LocRpt.cgi?l=3718
    ePath Adenosine deaminase (ADA); MIM number: 102700. OMIM: http://www.ncbi.nlm.nih.gov/htbin-post/Omim/dispmim?102700
    ePath Janus kinase 3 (a protein tyrosine kinase, leukocyte) (JAK3); MIM number: 600173. OMIM: http://www.ncbi.nlm.nih.gov/htbin-post/Omim/dispmim?600173

Related Sub-Topics:

Viruses in Gene Therapy | Specific Genetic Disorders | Techniques and Tools in Clinical Genetics | Gene Therapy
Contact Editor close
Submit a note to the editor about this article by filling in the form below.

* Required Field

Article Title: Immunodeficiency Syndromes: Gene Therapy
 
How to Cite close
Fischer, Alain(Jan 2006) Immunodeficiency Syndromes: Gene Therapy. In: eLS. John Wiley & Sons Ltd, Chichester. http://www.els.net [doi: 10.1038/npg.els.0005752]