1. ¹Department of Molecular Genetics and Biochemistry, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA
2. ²Department of Orthopaedic Surgery, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA
3. ³Department of Molecular Medicine Institute, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA
4. ⁴Department of Molecular Orthopaedics, Harvard Medical School, Boston, MA, USA
5. ⁵Bone and Joint Research Unit, St Bartholomew's and Royal London School of Medicine and Dentistry, Queen Mary University of London, UK

Correspondence: Dr PD. Robbins, Department of Molecular Genetics and Biochemistry, W1246 Biomedical Science Tower, University of Pittsburgh, School of Medicine, Pittsburgh, PA, USA

Abstract

Rheumatoid arthritis is an autoimmune disease with intra-articular inflammation and synovial hyperplasia that results in progressive degradation of cartilage and bone, in severe cases it causes systemic complications. Recently, biological agents that suppress the activities of proinflammatory cytokines have shown efficacy as antiarthritic drugs, but require frequent administration. Thus, gene transfer approaches are being developed as an alternative approach for targeted, more efficient and sustained delivery of inhibitors of inflammatory cytokines as well as other therapeutic agents. Indeed, the efficacy of gene transfer for the treatment of arthritis has been demonstrated in mouse, rat, rabbit, and horse models of disease whereas the feasibility of the approach has been demonstrated in Phase I clinical trials. In this review, the current status of both preclinical and clinical arthritis gene therapy is presented. In addition, the advantages and disadvantages of different types of vectors, target cells and therapeutic genes being developed for the treatment of arthritis are summarized. Finally, the future directions of the rapidly developed field of arthritis gene therapy are outlined