Immunodeficiency Syndromes: Gene Therapy


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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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:

Janus kinase 3 (a protein tyrosine kinase, leukocyte) (JAK3); Locus ID: 3718. LocusLink:

Adenosine deaminase (ADA); MIM number: 102700. OMIM:‐post/Omim/dispmim?102700

Janus kinase 3 (a protein tyrosine kinase, leukocyte) (JAK3); MIM number: 600173. OMIM:‐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. [doi: 10.1038/npg.els.0005752]