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.
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.
- IMDEA Nanociencia researchers use nanoparticles as vehicles to deliver microRNAs to cells and treat Duchenne Muscular Dystrophy.
- It is possible to specifically deliver the treatment to the muscle stem cells, avoiding accumulation in other organs.
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.
Álvaro Somoza, lead author of the study, is very enthusiastic about the results: “There are two very notable things to point out, first, the effective delivery of a microRNA to the desired organ, which increases the effectiveness of the therapy. On the other hand, this approach prevents the accumulation in other organs, such as the brain, kidneys or liver, which is key to prevent side effects.”.
