Study Suggests Gene Therapy May One Day Help with Muscle Development

Gene Therapy

A study published on May 8 in Science Advances by researchers at the Washington University School of Medicine in St. Louis suggested that gene therapy may be helpful for building strength and significant muscle mass quickly in the body.

The investigators drew their conclusions after studying gene therapy in mice. Senior investigator Farshid Guilak and his colleagues gave eight-week-old mice a single injection each of a virus carrying follistatin. Follistatin is a gene that works to block the activity of a protein in muscle that keeps growth in check.

Over time, the mice were able to gain significant muscle mass without exercising more than usual. They were also able to nearly double their strength, all while continuing to consume a high-fat diet. The mice exhibited less cartilage damage related to osteoarthritis, lower numbers of inflammatory cells and proteins in their joints, fewer metabolic problems, and healthier hearts and blood vessels compared to the control group.

Longer-term studies will be needed to determine if this gene therapy is safe. For instance, one concern is that some of the muscle growth may impact the heart by causing its walls to thicken. However, the researchers did not see any evidence of this in the mice.

“Something like this could take years to develop, but we’re excited about its prospects for reducing joint damage related to osteoarthritis, as well as possibly being useful in extreme cases of obesity,” said Guilak.

Many experts warn that strenuous activity can damage joints and be problematic for patients with osteoarthritis. However, a study published in JAMA by researchers at Northwestern Medicine on May 4 recently suggested that the development of radiographic knee osteoarthritis (KOA), in particular, is not linked to this type of physical activity. Overall, older adults at risk for the condition may even be able to improve their general health by engaging in strenuous physical activities at a moderate level.

To conduct their study, researchers looked at data from the Osteoarthritis Initiative, a prospective longitudinal cohort study of men and women with or at an increased risk of developing symptomatic, radiographic KOA. Adults were also included in the study from four different locations in the U.S., and followed for up to 10 years.

In the end, the investigators determined that the development of radiographic KOA could not be linked to long-term engagement in low-to-moderate physical activities or any strenuous physical activities.

“Results from this study appeared to show no association between long-term strenuous physical activity participation and incident radiographic KOA,” wrote the authors of the report. “The findings raise the possibility of a protective association between incident KOA and a low-to-moderate level of strenuous physical activities.”

The investigators also noted in their study that participation in exercise, sports or recreational activities can provide numerous health benefits and possibly increase quality of life.

Osteoarthritis is the most common type of arthritis, according to the Arthritis Foundation. When the cartilage between bones breaks down, joints can become painful, swollen and difficult to move. Osteoarthritis can impact any joint, but it most often appears in the hands, knees, hips, lower back and neck.

Medical history, a physical examination and lab tests can all be used to help make an osteoarthritis diagnosis. While there is no cure for osteoarthritis, medication and assistive devices can be used to ease pain in patients. Pain and anti-inflammatory medicines for the condition are available as pills, syrups, patches and creams, and they may even be injected into a problematic joint. Exercise is often recommended as an essential part of any treatment plan.

Back to news