Progress toward the treatment of arthritis by gene therapy

Gene therapy offers several novel advantages to the treatment of arthritis and other joint diseases. Therapeutic genes may be delivered locally to diseased joints or systemically to extra-articular locations using viral or non-viral vectors by ex vivo or in vivo strategies. Pre-clinical experiments with rabbits have demonstrated the utility of using a retrovirus to deliver the human interleukin-1 receptor antagonist to the synovial lining of joints. A human trial based on this principle has been approved by the Recombinant DNA Advisory Committee of NIH and should begin this year.