Gene therapy in autoimmune diseases: Challenges and opportunities

Abstract Clinical treatment of autoimmune disorders presents a special challenge. For decades, most clinical regimens in autoimmunity has been largely symptomatic and non-disease specific. Although data from vigorous research has lead to accumulating knowledge on the pathogenic and immunological mechanisms of many autoimmune diseases, their direct clinical applications have been sparse. Advances in biotechnology have laid the groundwork for potent and specific molecular targeting therapies by gene therapy, and have just begun to be investigated in the treatment of autoimmune disorders. Such work has been largely based on the availability of well-established animal models of common autoimmune disorders, and the efficacy of strategic approaches initially investigated and validated in these models. Although these preclinical animal model studies have provided the proof-of-concept for multiple potential applications, human clinical trials on gene therapy in autoimmunity are still at its infancy. The recent success of Phase I/II clinical trials of gene therapy in rheumatoid arthritis and multiple sclerosis, development of cutting edge technology in target identification, as well as gene delivery systems have now set the stage for a more thorough and vigorous pace in the near future to advance this exciting field.