Mechanisms and applications of stem cell gene therapy in autoimmunity

Autoimmune diseases are the manifestation of an aberrant immune system that targets our own tissues and affects 5–6% of the population. Current therapies are largely dependent on replacement therapy and/or long-term, toxic immunosuppression. Clinical trials of autologous bone marrow transplantation as curative therapy are underway but appear limited by high relapse rates. Experimental animal studies suggest that genetically manipulated hematopoietic stem cells have the potential of providing a cure without the risk of relapse. This review will focus on such an approach in which causative self-antigens are known.

Section editors:David Scott – University of Mariland School of Medicine, Baltimore, USATerry L. Delovitch – The John P. Robarts Research Institute, London, Canada