Progress and Prospects: Genetic treatments for disorders of bones and joints

Gene therapies directed toward the treatment of arthritis and tissue repair continue to be the most active areas of research for bone and joint diseases. In the past 2 years, two trials in rheumatoid arthritis have been completed. a Phase I study reporting safety and a Phase I/II study that has yet to be published. An additional, small study has reported the first evidence of clinical efficacy. Two Phase I trials of gene therapy for osteoarthritis have also been initiated. There is much preclinical activity in developing AAV vectors for future trials in the gene therapy of arthritis. Research into tissue repair and regeneration remains at the preclinical stage, but a considerable volume of research attests to the promise of gene transfer in this arena, especially in the context of bone healing. For tissue repair, the major research questions are still which genes to use and how best to deliver them.