Gene Therapy for Cartilage Regeneration

Christopher H. Evans, Ph.D., director of the Rehabilitation Medicine Research Center at Mayo Clinic's campus in Rochester, Minnesota, and Carmen M. Terzic, M.D., Ph.D., chair of Physical Medicine and Rehabilitation, discuss gene therapy for cartilage regeneration at the 2015 Fourth Annual Symposium on Regenerative Rehabilitation.

Cartilage is unusual tissue because it is one of the few organs in the body that cannot repair itself after injury. Cartilage is frequently injured in sporting activities and accidents and is damaged by diseases such as arthritis.

It is important to be inventive when developing ways to repair cartilage, notes Dr. Evans. The Rehabilitation Medicine Research Center is working 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 administer a single shot that will work over the weeks or 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 follow.


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