Autoimmune Diseases: Gene Therapy


Gene therapy is a method of reconstituting or altering gene expression that can be used to correct genetic defects or inappropriate immune responses that occur in autoimmune diseases. The efficacy of this approach can be successfully tested in animal models of autoimmune diseases prior to application in human autoimmune disease.

Keywords: autoimmunity; gene therapy; animal model; clinical trial; rheumatoid arthritis


Chen Y, Donnelly E, Kobayashi H, Debusk LM and Lin PC (2005) Gene therapy targeting the Tie2 function ameliorates collagen‐induced arthritis and protects against bone destruction. Arthritis and Rheumatism 52(5): 1585–1594.

Evans CH, Robbins PD, Ghivizzani SC et al. (2005) Gene transfer to human joints: Progress toward a gene therapy of arthritis. Proceedings of the National Academy of Sciences of the USA 102(24): 8698–8703.

Irie J and Ridgway WM (2005) A modular theory of autoimmunity. Keio Journal of Medicine 54(3): 121–126.

Loser K, Hansen W, Apelt J et al. (2005) In vitro generated regulatory T cells induced by Foxp3‐retrovirus infection control murine contact allergy and systemic autoimmunity. Gene Therapy 12(17): 1294–1304.

McInnes IB and Liew FY (2005) Cytokine networks – towards new therapies for rheumatoid arthritis. Nature Clinical Practice Rheumatology 1(1): 31–39.

Neumann E, Judex M, Kullmann F et al. (2002) Inhibition of cartilage destruction by double gene transfer of IL‐1Ra and IL‐10 involves the activin pathway. Gene Therapy 9(22): 1508–1519.

Sapir T, Shternhall K, Meivar‐Levy L et al. (2005) Cell‐replacement therapy for diabetes: generating functional insulin‐producing tissue from adult human liver cells. Proceedings of the National Academy of Sciences of the USA 102(22): 7964–7969.

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Tarner IH, Müller‐Ladner U and Fathman CG (2004) Targeted gene therapy: frontiers in the development of ‘smart drugs’. Trends in Biotechnology 22(6): 304–310.

Tas SW, Adriaansen J, Hajji N et al. (2006) Amelioration of arthritis by intraarticular dominant negative IKKβ gene therapy using adeno‐associated virus type 5. Human Gene Therapy 17(8): 821–832.

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Zhang H, Gao G, Clayburne G and Schumacher HR (2005) Elimination of rheumatoid synovium in situ using a Fas ligand ‘gene scalpel’. Arthritis Research and Therapy 7(6): 1235–1243.

Further Reading

Browning JL (2006) B cells move to centre stage: novel opportunities for autoimmune disease treatment. Nature Reviews Drug Discovery 5(7): 564–576.

Hacein‐Bey‐Abina S, von Kalle C, Schmidt M et al. (2003) A serious adverse event after successful gene therapy for X‐linked severe combined immunodeficiency. New England Journal of Medicine 348(3): 255–256.

Hacein‐Bey‐Abina S, von Kalle C, Schmidt M et al. (2003) LMO2‐associated clonal T cell proliferation in two patients after gene therapy for SCID‐X1. Science 302(5644): 415–419.

O’Neill LAJ (2006) Targeting signal transduction as a strategy to treat inflammatory diseases. Nature Reviews Drug Discovery 5(7): 549–563.

Peng Z (2005) Current Status of Gendicine in China: Recombinant human Ad‐p53 agent for treatment of cancers. Human Gene Therapy 16(9): 1016–1027.

Prud’homme GJ, Glinka Y, Khan AS and Draghia‐Akli R (2006) Electroporation‐enhanced nonviral gene transfer for the prevention or treatment of immunological, endocrine and neoplastic diseases. Current Gene Therapy 6(2): 243–273.

Redlich K, Schett G, Steiner G et al. (2003) Rheumatoid arthritis therapy after tumor necrosis factor and interleukin‐1 blockade. Arthritis and Rheumatism 48(12): 3308–3319.

Somia N and Verma IM (2000) Gene therapy: trials and tribulations. Nature Reviews Genetics 1(2): 91–99.

Web links

Searchable database for clinical trials in the United States.

Website of the US National Institute for Allergy and Immune diseases with downloadable material for the general as well as scientific reader.

Part of the Website of the Human Genome Project outlining the possibilities of gene therapy.

Most comprehensive source for clinical trials of gene therapy worldwide.

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How to Cite close
Purath, Ulrich, Tarner, Ingo H, and Fathman, C Garrison(Jan 2007) Autoimmune Diseases: Gene Therapy. In: eLS. John Wiley & Sons Ltd, Chichester. [doi: 10.1002/9780470015902.a0005759.pub2]