Gene therapy in the musculoskeletal system

Purpose of review: This review summarizes recent progress in applying gene therapy to the musculoskeletal system. Recent findings: Most progress has been made with gene therapy for arthritis, for which four phase I clinical trials have been initiated and a phase II protocol has been developed. Preliminary studies support the use of gene transfer approaches in the treatment of other chronic conditions, including osteoporosis and aseptic loosening. In the immediate future, however, most rapid progress is likely to come from applications in tissue repair, because they need neither long-term transgene expression nor, in most cases, closely regulated levels of transgene expression. There is a rapidly growing literature on the use of gene transfer to aid the repair of bone, articular cartilage, ligament, tendon, meniscus, and intervertebral disk. In contrast, the possible use of gene therapy to treat orthopedic tumors has received scant attention. Summary: Recent developments in orthopedic gene therapy are highly encouraging. Gene therapy has the potential to transform many areas of orthopedic care, ultimately leading to solutions for pressing clinical problems and to treatments which are less invasive, cheaper, and more effective than existing modalities.