Gene therapy in rheumatic diseases.

Abstract

Rheumatoid arthritis (RA) is a chronic inflammatory disease characterized by joint inflammation as well as progressive cartilage and bone destruction. Advances in the understanding of the pathophysiology of RA have led to the development of new therapeutic strategies, including gene therapy. Gene therapy offers a new approach to deliver therapeutic proteins to the joints of arthritis patients. Local as well as systemic gene therapy can be envisaged for the treatment of arthritis. Several viral and non-viral vectors have been used in animal models for rheumatoid arthritis for ex vivo and in vivo delivery of therapeutic genes. Promising pre-clinical data have resulted from the application of these strategies. Using ex vivo gene delivery, successful and safe gene transfer has been demonstrated in the joints of RA patients. Although new insights into the role of cytokines and other mediators of chronic inflammation have provided novel targets for therapeutic intervention, the development of vectors that induce long-term and regulated gene expression remains a challenge.