Gene Therapy for Autoimmune Disease

Shang An Shu, Jinjun Wang, Mi Hua Tao, Patrick S Leung

Research output: Contribution to journalArticlepeer-review

14 Scopus citations


Advances in understanding the immunological and molecular basis of autoimmune diseases have made gene therapy a promising approach to treat the affected patients. Gene therapy for autoimmune diseases aims to regulate the levels of proinflammatory cytokines or molecules and the infiltration of lymphocytes to the effected sites through successful delivery and expression of therapeutic genes in appropriate cells. The ultimate goal of gene therapy is to restore and maintain the immune tolerance to the relevant autoantigens and improve clinical outcomes for patients. Here, we summarize the recent progress in identifying genes responsible for autoimmune diseases and present examples where gene therapy has been applied as treatments or prevention in autoimmune diseases both in animal models and the clinical trials. Discussion on the advantages and pitfalls of gene therapy strategies employed is provided. The intent of this review is to inspire further studies toward the development of new strategies for successful treatment of autoimmune diseases.

Original languageEnglish (US)
Pages (from-to)163-176
Number of pages14
JournalClinical Reviews in Allergy and Immunology
Issue number2
StatePublished - Oct 3 2014


  • Animal model
  • Autoimmune disease
  • Clinical trial
  • Delivery vectors
  • Gene therapy

ASJC Scopus subject areas

  • Immunology and Allergy


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