A new strategy for the treatment of Duchenne Muscular Dystrophy

The results of the research published on Nature Nanotechnology
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muscle in a magnetic field and exosomes injection

Dr. Valeria Secchi, Dr. Michele Mauri, Prof. Roberto Simonutti and Prof. Angelo Monguzzi, researchers of the Department of Materials Science, University of Milano-Bicocca in collaboration with the research group of Dr. Chiara Villa and Prof. Yvan Torrente of the Stem Cell Laboratory, “Centro Dino Ferrari,” Department of Medical and Surgical Pathophysiology and Transplantation, University of Milan, Neurology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, and the group of Prof. Domenico Aquino of the Department of Neuroradiology of the Carlo Besta Institute in Milan, have discovered a new strategy for the treatment of Duchenne Muscular Dystrophy (DMD).

The subject of this research is exosomes, nanovesicles that can promote tissue repair and regeneration. “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,” says Dr. Valeria Secchi. She also adds : “The resolution of inflammation in the context of muscular dystrophies is one of the most difficult challenges that researchers and clinicians try to solve”.

The authors of this study have developed a novel method to deliver engineered exosomes with anti-inflammatory properties into dystrophic muscles. Using ferromagnetic nanotubes as the carrier, these exosomes can migrate specifically into damaged muscles by application of an external magnetic field after systemic injection.

Quantitative analyses at the muscle level revealed that uptake of injected exosomes promotes regeneration and improves muscle performance in a mouse model of Duchenne Muscular Dystrophy. “The results obtained provide new insights,” says Prof. Angelo Monguzzi, ”for the development of therapies based on natural and synthetic nanovesicles in order to treat different forms of muscle diseases. In general, they highlight the importance of the design and formulation of effective advanced multifunctional nanovectors aimed at optimizing the biodistribution of nanovesicles”.

This discovery represents a significant breakthrough in the field of regenerative medicine and innovative therapies for muscle diseases. Precise control of exosome biodistribution opens up new possibilities for treating not only Duchenne Muscular Dystrophy, but also other muscle diseases characterized by chronic inflammation and tissue degeneration. The combination of ferromagnetic nanotubes and engineered exosomes could revolutionize therapeutic approaches, offering real hope for patients and families affected by these debilitating diseases.

The results of the research have been reported in the paper "Magnetic-field-driven targeting of exosomes modulates immune and metabolic changes in dystrophic muscle"  (DOI: s41565-024-01725-y), recently published on Nature Nanotechnology (Impact Factor 38.1, 2023 Journal Impact Factor, Journal Citation Reports, Clarivate Analytics, 2024).