In a collaboration between the IMDEA Nanociencia Institute (Madrid), Università Cattolica del Sacro Cuore (Rome), and the University of Bordeaux, 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.
MicroRNA is a class of RNA molecules that play a crucial role in gene regulation. The delivery of microRNAs through the bloodstream is complicated, due to low stability and penetration. In this context, nanoparticles act as safe biocarriers and improve the delivery of this therapy.
In their work, the researchers developed an aptamer, a molecule that selectively recognizes specific other molecules, in this case, proteins that are in muscle stem cells. By combining the aptamer with a nanoparticle, they were able to release the microRNA on muscle stem cells with great precision, and reactivate muscle regeneration.
Researchers have reported the activity of the system in cellular and animal models, where they have observed not only muscle regeneration at the cellular level, but recovery at the functional level. The muscles of the treated mice improved, they were stronger after the treatment, and thus enhanced the mice’s functional capacity.
Complete study at:
