Gene Therapy allows for muscle growth.

From movie stars to athletes to the average highschool teen, the desire to have a muscular body has become ever more important. Outside of popular culture, several neuromuscular disorders result in severe weakness of the quadriceps. Researchers at Nationwide Children's Hospital are developing a gene delivery strategy that produces follistatin. Follistatin is a naturally occurring protein that inhibits myostatin, a growth factor expressed specifically in skeletal muscle. The result of this inhibition is that directly long-term gene expression with muscle enhancing effects.

Brian Kaspar, PhD, principal investigator in the Center for Gene Therapy of The Research Institute at Nationwide Children's Hospital said that "studies indicate that this relatively non-invasive approach could have long-term effects, involve few risks and could potentially be effective in various types of degenerative muscle disorders including multiple forms of muscular dystrophy." Their research has proven successful in non-human primates. The muscles size was shown to increase by 15%, and muscle strength was shown to increase by 78%. The effect lasted for the 15 month study with no negative health effects. The research could also highlight methods of weight control and reduction of obesity.


This drug would be able to tap into an extensive market with an estimated 7 million people who take steroids to boost their muscles (in spite of the health risks), while also providing a solution to a wide range of neuromuscular diseases and weight control. Thus, the research has the potential, like many other gene targeted drugs, to remove a large burden from the current healthcare infrastructure. Shifting emphasis towards preventive solutions, and avoiding high cost surgical and invasive options.