Duchenne Dystrophy, an Italian Way to Repair and Regenerate Muscles

From Italian research a new strategy to promote muscle repair and regeneration in Duchenne dystrophy patients. The discovery, published in ‘Nature Nanotechnology’, bears the signature of a team of scientists from Milan. The authors have developed an innovative method to specifically deliver engineered microvesicles (exosomes) with anti-inflammatory properties into dystrophic muscle tissue.

The research is coordinated by Chiara Villa and Yvan Torrente of the Stem Cell Laboratory, Dino Ferrari Center, of the Department of Medical-Surgical Physiopathology and Transplants of the State University of Milan and of the Neurology Unit of the Policlinico city hospital, in collaboration with the groups of Angelo Monguzzi (Department of Materials Science of the University of Milan-Bicocca) and Domenico Aquino (Department of Neuroradiology of the IRCCS Carlo Besta Neurological Institute of Milan).

“Resolving inflammation in the context of muscular dystrophies is one of the most difficult challenges that researchers and clinicians are trying to solve,” explains Villa. Scientists have found a way to ‘target’ engineered exosomes with anti-inflammatory action in diseased muscles: using ferromagnetic nanotubes as a carrier, these exosomes can migrate specifically into muscles damaged by Duchenne muscular dystrophy (DMD) through the application of an external magnetic field after a systemic injection. “We were able to control the biodistribution and targeting of exosomes in vivo in order to reduce the inflammatory condition of muscles affected by DMD,” Villa emphasizes. Quantitative analyses at the muscle level showed that macrophages, a type of white blood cell, dominate the uptake of injected exosomes, promoting muscle regeneration and improving muscle performance in a mouse model of Duchenne muscular dystrophy.

“Our results – comments Torrente – provide new insights for the development of therapies based on natural and synthetic microvesicles in order to treat different forms of muscle diseases. In general, they highlight the formulation of effective functional nanovectors aimed at optimizing the biodistribution of microvesicles”.

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This discovery, as stated in a UniMi note, represents “a significant step forward in the field of regenerative medicine and innovative therapies for muscle diseases. The precise control of the biodistribution of exosomes opens” in fact “new possibilities for the treatment not only of Duchenne muscular dystrophy, but also of other muscle diseases characterized by chronic inflammation and tissue degeneration. The combination of ferromagnetic nanotubes and engineered exosomes could revolutionize the therapeutic approach, offering concrete hope for patients and families affected by these debilitating diseases”.