Gene therapy for autoimmune disorders

Christopher H Evans, S. C. Ghivizzani, T. J. Oligino, P. D. Robbins

Research output: Contribution to journalArticle

24 Citations (Scopus)

Abstract

Although many autoimmune disorders do not have a strong genetic basis, their treatment may nevertheless be improved by gene therapies. Most strategies seek to transfer genes encoding immunomodulatory products that will alter host immune responses in a beneficial manner. Used in this fashion, genes serve as biological delivery vehicles for the products they encode. By this means gene therapy overcomes obstacles to the targeted delivery of proteins and RNA, and improves their efficacy while providing a longer duration of effect, and, potentially, greater safety. Additional genetic strategies include DNA vaccination and the ablation of selected tissues and cell populations. There is considerable evidence from animal studies that gene therapies work: examples include the treatment of experimental models of rheumatoid arthritis, multiple sclerosis, diabetes, and lupus. Pre-clinical success in treating animal models of rheumatoid arthritis has led to the first clinical trial of gene therapy for an autoimmune disease. In this Phase I study, a cDNA encoding the interleukin-1 receptor antagonist was transferred to the knuckle joints of patients with advanced rheumatoid arthritis. Two additional clinical trials are in progress. It is likely that gene therapy will provide effective new treatments for a wide range of autoimmune disorders.

Original languageEnglish (US)
Pages (from-to)334-346
Number of pages13
JournalJournal of Clinical Immunology
Volume20
Issue number5
DOIs
StatePublished - 2000
Externally publishedYes

Fingerprint

Genetic Therapy
Rheumatoid Arthritis
Clinical Trials
Interleukin-1 Receptors
Genes
Autoimmune Diseases
Multiple Sclerosis
Vaccination
Theoretical Models
Therapeutics
Animal Models
Complementary DNA
Joints
RNA
Safety
DNA
Population
Proteins

Keywords

  • Cytokine
  • Dendritic cell
  • Gene
  • Human trial
  • Synovium
  • T-lymphocyte
  • Vector

ASJC Scopus subject areas

  • Immunology
  • Immunology and Allergy

Cite this

Evans, C. H., Ghivizzani, S. C., Oligino, T. J., & Robbins, P. D. (2000). Gene therapy for autoimmune disorders. Journal of Clinical Immunology, 20(5), 334-346. https://doi.org/10.1023/A:1006616029205

Gene therapy for autoimmune disorders. / Evans, Christopher H; Ghivizzani, S. C.; Oligino, T. J.; Robbins, P. D.

In: Journal of Clinical Immunology, Vol. 20, No. 5, 2000, p. 334-346.

Research output: Contribution to journalArticle

Evans, CH, Ghivizzani, SC, Oligino, TJ & Robbins, PD 2000, 'Gene therapy for autoimmune disorders', Journal of Clinical Immunology, vol. 20, no. 5, pp. 334-346. https://doi.org/10.1023/A:1006616029205
Evans, Christopher H ; Ghivizzani, S. C. ; Oligino, T. J. ; Robbins, P. D. / Gene therapy for autoimmune disorders. In: Journal of Clinical Immunology. 2000 ; Vol. 20, No. 5. pp. 334-346.
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