The Musculoskeletal Gene Therapy Research Laboratory at Mayo Clinic is leading innovative ways to improve treatment for debilitating joint and bone conditions, using gene therapy as the main technology platform.

Led by principal investigator Christopher H. Evans, Ph.D., our lab aims to solve clinical problems in orthopedics using gene transfer, through a focus on two broad areas of research:

  • Joint diseases such as osteoarthritis
  • Tissue regeneration after musculoskeletal injuries

Osteoarthritis research

Osteoarthritis is a major focus of our research on joint diseases. That's because more than 27 million people in the United States have osteoarthritis, but it remains incurable and very difficult to treat effectively. Osteoarthritis is a source of much misery and generates more than $100 billion a year in health care costs.

Our lab is studying gene therapy as a promising potential treatment for osteoarthritis. We're running a phase I clinical trial that involves injecting an attenuated virus carrying an anti-arthritic gene into the knee joints of patients with osteoarthritis. Each participant in the trial is monitored for one year to assess safety, dosing and, in a preliminary manner, efficacy.

Musculoskeletal injury research

Like osteoarthritis, musculoskeletal injuries are very common. Risk factors for musculoskeletal injuries include overuse, aging, trauma, metabolic disorders and obesity. Different tissues vary in their ability to heal spontaneously from musculoskeletal injuries.

Bone fractures, for example, typically heal by themselves, except when large segments of bone have been removed or when there is substantial damage to surrounding soft tissues. Tendons and many ligaments are able to heal by themselves, too, but the regenerated tissue is of inferior quality and prone to re-injury. In contrast, cartilage has little or no intrinsic ability to repair itself.

Our lab is engaged in preclinical research in all these areas of musculoskeletal injury, using animal models. In addition to exploring the use of traditional, virally mediated gene transfer, we are investigating the use of chemically modified RNA in collaboration with investigators from the Technical University of Munich in Munich, Germany.

About Dr. Evans

In addition to leading the Musculoskeletal Gene Therapy Research Lab, Dr. Evans is the director of the Rehabilitation Medicine Research Center at Mayo Clinic. He also is a professor of orthopedics and of physical medicine and rehabilitation at Mayo Clinic College of Medicine and Science in Rochester, Minnesota. He is also Dr. Evans hopes that his research on arthritis and tissue regeneration will lead to improvements in treatment options for people with osteoarthritis and bone and cartilage damage.