Development and validation of gene therapies in autoimmune diseases: Epidemiology to animal models

Patrick S Leung, Shang An Shu, Thomas P. Kenny, Ping Yi Wu, Mi Hua Tao

Research output: Contribution to journalArticlepeer-review

30 Scopus citations


Recent advancement in immunology, molecular biology, and bioinformatics has yielded extensive information on the pathophysiological mechanisms of autoimmunity, which has greatly facilitated the identification of potential therapeutic targets and the development of gene therapy in the treatment of autoimmune disease. Preclinical studies were carried out in animal models. This phenomenon is well illustrated in two prototypic animal models of autoimmune disease: the autoimmune encephalomyelitis (EAE) model of multiple sclerosis (MS) and collagen-induced arthritis (CIA) model in rheumatoid arthritis (RA). Here we discuss the current data on the development and validation of gene therapy in autoimmunity in these two models. The success in preclinical animal model studies provides the proof-of-concept of gene therapy for potential future applications in the treatment of autoimmune diseases. Furthermore, the identification of risk factors from epidemiological studies reveals further potential therapeutic targets to be examined in animal models.

Original languageEnglish (US)
JournalAutoimmunity Reviews
Issue number5
StatePublished - Mar 2010


  • Collagen-induced arthritis
  • Epidemiology
  • Experimental autoimmune encephalomyelitis
  • Gene therapy
  • Multiple sclerosis
  • Rheumatoid arthritis

ASJC Scopus subject areas

  • Immunology
  • Immunology and Allergy


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