New Muscle-Wasting Gene Therapy Studied

U.S. medical scientists have identified the role of a protein that might lead to new treatments for musculoskeletal diseases, such as muscular dystrophy.

Researchers at Nationwide Children’s Hospital said the findings show promise for development of potential new gene therapy strategy for muscle-wasting diseases.

The research, led by Ohio State University Assistant Professor Brian Kaspar, focused on a protein called follistatin, or FS. Using a single injection, gene-delivery strategy, investigators treated the hind leg muscles of mice. Results showed increased muscle size and strength — quadruple that of mice treated with proteins other than FS. The muscle enhancements were shown to be well-tolerated for more than two years.

We believe this new FS strategy may be more powerful than other strategies due to its additional effects, including its ability to reduce inflammation, said Kaspar.

The researchers said the strategy showed no negative effects on the heart or reproductive ability of either males or females. The results were also replicated in older animals, suggesting the strategy could be useful in developing clinical treatments for older patients.

The study is reported in the Proceedings of the National Academy of Sciences.