Immunodeficiency Syndromes: Gene Therapy

Abstract

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.

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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 β‐thalassemic mice expressing lentivirus‐encoded human β‐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

Adenosine deaminase (ADA); Locus ID: 100. LocusLink: http://www.ncbi.nlm.nih.gov/LocusLink/LocRpt.cgi?l=100

Janus kinase 3 (a protein tyrosine kinase, leukocyte) (JAK3); Locus ID: 3718. LocusLink: http://www.ncbi.nlm.nih.gov/LocusLink/LocRpt.cgi?l=3718

Adenosine deaminase (ADA); MIM number: 102700. OMIM: http://www.ncbi.nlm.nih.gov/htbin‐post/Omim/dispmim?102700

Janus kinase 3 (a protein tyrosine kinase, leukocyte) (JAK3); MIM number: 600173. OMIM: http://www.ncbi.nlm.nih.gov/htbin‐post/Omim/dispmim?600173

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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]