About the Lab

The Musculoskeletal Gene Therapy Research Laboratory at Mayo Clinic's campus in Rochester, Minnesota, aims to solve clinical problems in orthopedics using gene transfer.

The Musculoskeletal Gene Therapy Research Lab is led by Christopher H. Evans, Ph.D.

There are two broad areas of research interest in Dr. Evans' lab:

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

Osteoarthritis research

Osteoarthritis is a major focus of the lab's research on joint diseases.

More than 27 million Americans have osteoarthritis, which is 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.

Under the direction of Dr. Evans, the Musculoskeletal Gene Therapy Research Lab is advancing gene therapy research for osteoarthritis. The lab has approval from the Food and Drug Administration for a clinical trial on gene therapy for osteoarthritis. This phase I clinical trial involves the injection of an attenuated virus carrying an anti-arthritic gene into the knee joints of patients with osteoarthritis. Each patient 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 these 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.

The Musculoskeletal Gene Therapy Research 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, the lab recently started research about the use of chemically modified RNA in conjunction with investigators from the Technical University of Munich in Munich, Germany.