Gene Therapy for Cartilage Regeneration

Christopher H. Evans, Ph.D., Director of Mayo Clinic Rehabilitation Medicine Research Center, Professor of Orthopedics and Carmen Terzic, M.D., Ph.D., Chair of Mayo Clinic Physical Medicine and Rehabilitation discuss Gene Therapy for Cartilage. Cartilage is unusual tissue because it’s one of the few organs in the body that has no ability to repair itself after injury. It is frequently injured in sporting activities and accidents, and is damaged by diseases such as arthritis. It’s important to be inventive when developing ways to repair cartilage. The approach the RMRC uses is to develop technologies that can be implemented in the operating room in a single procedure; nothing has to leave or be grown outside the body. Gene transfer is used in a single surgery without the need to groom the cells. Surgeons will administer a single shot that will work over the weeks and months it takes to create new cartilage. When a gene is placed in the cells, the instructions present in that gene will remain with the cells and produce the sought-after effect. This procedure is currently being performed in animal models, and if successful, human clinical trials will be next.

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