In an international collaboration, researchers have made an important breakthrough in the therapeutic delivery of microRNAs against Duchenne muscular dystrophy, a disease with no cure, to date.

Duchenne muscular dystrophy is a genetic disorder characterized by the progressive loss of muscle mass, due to mutations in the dystrophin gene. Without the corresponding functional protein, muscles cannot function or repair themselves properly, resulting in the deterioration of skeletal, heart, and lung muscles. Because the dystrophin gene is located on the X chromosome, it mainly affects males, while females are usually carriers.

Researchers have developed a strategy to treat muscular dystrophy, which uses nanoparticles as vehicles to transport therapeutical microRNAs to muscle stem cells. Once inside the muscle stem cells, the nanoparticles release the microRNA to stimulate the production of muscle fibers.