Gene Therapy Can Improve Muscle Mass and Strength in Monkeys
A study appearing in Science Translational Medicine puts scientists one step closer to clinical trials to test a gene delivery strategy to improve muscle mass and function in patients with certain degenerative muscle disorders.
Severe weakness of the quadriceps is a defining feature of several neuromuscular disorders. Researchers at Nationwide Children’s Hospital have shown that a gene delivery strategy that produces follistatin – a naturally occurring protein that inhibits myostatin, a growth factor expressed specifically in skeletal muscle – directly to the quadriceps of non-human primates results in long-term gene expression with muscle enhancing effects, including larger muscles with greater strength.
