Despite setbacks in other fields, gene therapy in orthopaedic surgery continues to serve as the basis for novel treatments of various musculoskeletal disorders. Even in the brief time since the last review of scientific progress in this area, another orthopaedic-related disease has joined the ranks of those studied in gene therapy clinical trials. Armed with new techniques and new reagents, and committed to the increased use of tissue engineering, physicians and scientists continue to work together to accelerate tissue repair and reverse the course of chronic debilitating diseases.