The Community of Madrid has achieved significant progress in therapy for Duchenne muscular dystrophy, a degenerative disease for which there is still no cure. The Madrid Institute for Advanced Studies (IMDEA Nanoscience), located in the capital, in collaboration with the Catholic University of the Sacred Heart in Rome and the University of Bordeaux, has developed a strategy that allows for the more effective transport of microRNA (molecules that regulate genes) and the stimulation of fiber production.
New Research for Duchenne Muscular Dystrophy
Researchers have developed a strategy that uses nanoparticles as vehicles to precisely deliver this therapy to stem cells, an advance that also prevents its accumulation in other organs such as the brain, kidneys, or liver.
This genetic disorder is characterized by progressive muscle loss due to mutations in a gene that regulates a protein necessary for proper function and regeneration. People who suffer from it experience deterioration of their skeletal, cardiac, and pulmonary muscles.
There are currently numerous clinical trials attempting to treat the disease through intravenous drug administration. However, delivery through the bloodstream is complicated by the drug’s poor stability and penetration.
Promising Results
Thanks to this, cellular and biochemical changes have been achieved in laboratory and animal models, where not only regeneration at the cellular level but also recovery at the functional level have been observed. Furthermore, this method, developed by Professor Álvaro Somoza’s team at IMDEA Nanoscience, is biocompatible, nontoxic, and nonimmunogenic, and can be easily adapted to address various diseases.
This technique, demonstrated so far in animal models, is not yet ready for use in humans. However, it paves the way for the development of future treatments that, leveraging this knowledge, can effectively treat Duchenne muscular dystrophy.
Learn More: New Gene Therapies for Duchenne Muscular Dystrophy
