Overview

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 conditions such as osteoarthritis.
  • Tissue regeneration after musculoskeletal injuries.

Osteoarthritis research

Osteoarthritis is a major focus of our research on joint conditions. It remains incurable and difficult to treat effectively. Our lab is studying gene therapy as a promising potential treatment for osteoarthritis.

We recently completed a phase 1 clinical trial on osteoarthritis. This trial involved injecting an attenuated virus carrying an antiarthritic gene into the knee joints of people with osteoarthritis. The trial data confirm that this gene therapy is safe and successfully delivers the antiarthritic gene to joints with osteoarthritis. We're now expanding to a larger phase 1b trial of this gene therapy. Read more about our osteoarthritis gene therapy trial.

Regeneration of musculoskeletal tissues

Like osteoarthritis, musculoskeletal injuries are also common. Risk factors for musculoskeletal injuries include trauma, overuse, aging, metabolic disorders and obesity.

Different musculoskeletal tissues vary in their ability to heal spontaneously after injury. Bone fractures, for example, typically heal by themselves, except when large segments of bone have been removed or when there's 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 reinjury. 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. We're making particular progress on bone healing with research funding from the U.S. National Institutes of Health (NIH). We're exploring the use of a gene therapy that delivers a bone-forming gene into fracture sites. We recently found that co-delivery of an anti-inflammatory gene greatly improves healing. In addition to exploring the use of traditional, virally mediated gene transfer, we're investigating the use of chemically modified messenger RNA. Read more about our musculoskeletal research.

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 is the John and Posy Krehbiel Professor of Orthopedics. He is a professor of physical medicine and rehabilitation and a professor of orthopedics at Mayo Clinic College of Medicine and Science in Rochester, Minnesota. Dr. Evans hopes that his research on arthritis and tissue regeneration will lead to improvements in treatment options for people with osteoarthritis and damaged bone, cartilage and tendons.