ArticleMolecular therapy : the journal of the American Society of Gene Therapy2021
Exosome-mediated improvement in membrane integrity and muscle function in dystrophic mice.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.
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Who cites it
25 citing papers in PubMed, 31 citations in OpenAlex.
- Exosomes target liver TREM-1 to break the systemic inflammatory cascade and improve spinal cord injury outcomes.Journal of orthopaedic translation · 2026Article
- Skeletal muscle‑derived extracellular vesicles in multi‑organ degenerative disease: Mechanisms and therapeutic delivery perspectives (Review).International journal of molecular medicine · 2026Review
- Engineered exosomes improve myocardial cell membrane integrity and heart function in dystrophic cardiomyopathy.Clinical and translational medicine · 2026Article
- Transforming Duchenne muscular dystrophy therapy: The multifaceted role of extracellular vesicles and exosomes.Biochemistry and biophysics reports · 2026Review
- Exosome Augmentation Technologies for Drug Delivery and Disease Treatment: A Review.Biomaterials research · 2026Review
- Novel Translational Concept: Axon-to-Muscle Exosomal Signaling as an Emerging Therapeutic Target in Spinal Muscular Atrophy.Biomedicines · 2025Review
- Engineering Targeted Gene Delivery Systems for Primary Hereditary Skeletal Myopathies: Current Strategies and Future Perspectives.Biomedicines · 2025Review
- Plasma Microvesicles May Contribute to Muscle Damage in theInternational journal of molecular sciences · 2025Article
- Liver-Secreted Extracellular Vesicles Promote Cirrhosis-Associated Skeletal Muscle Injury Through mtDNA-cGAS/STING Axis.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Article
- Recent Advances of Exosomes Derived from Skeletal Muscle and Crosstalk with Other Tissues.International journal of molecular sciences · 2024Review
- Proteomic profiling of human plasma extracellular vesicles identifies PF4 and C1R as novel biomarker in sarcopenia.Journal of cachexia, sarcopenia and muscle · 2024Article
- In Vitro Studies to Evaluate the Intestinal Permeation of an Ursodeoxycholic Acid-Conjugated Oligonucleotide for Duchenne Muscular Dystrophy Treatment.Pharmaceutics · 2024Article
- Generalizable anchor aptamer strategy for loading nucleic acid therapeutics on exosomes.EMBO molecular medicine · 2024Article
- Challenges and Considerations of Preclinical Development for iPSC-Based Myogenic Cell Therapy.Cells · 2024Review
- Duchenne muscular dystrophy treatment with lentiviral vector containing mini-dystrophin gene in vivo.MedComm · 2024Article
- Selective Delivery to Cardiac Muscle Cells Using Cell-Specific Aptamers.Pharmaceuticals (Basel, Switzerland) · 2023Article
- Exercise-derived skeletal myogenic exosomes as mediators of intercellular crosstalk: a major player in health, disease, and exercise.Journal of physiology and biochemistry · 2023Review
- Lipin1 plays complementary roles in myofibre stability and regeneration in dystrophic muscles.The Journal of physiology · 2023Article
- Exosome-mediated regulatory mechanisms in skeletal muscle: a narrative review.Journal of Zhejiang University. Science. B · 2023Review
- Extracellular vesicles and Duchenne muscular dystrophy pathology: Modulators of disease progression.Frontiers in physiology · 2023Review
Corrections and comments
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Authors and funding
11 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Duchenne muscular dystrophy (DMD) is a devastating genetic disorder that leads to compromised cellular membranes, caused by the absence of membrane-bound dystrophin protein. Muscle membrane leakage results in disrupted intracellular homeostasis, protein degradation, and muscle wasting. Improving muscle membrane integrity may delay disease progression and extend the lifespan of DMD patients. Here, we demonstrate that exosomes, membranous extracellular vesicles, can elicit functional improvements in dystrophic mice by improving muscle membrane integrity. Systemic administration of exosomes from different sources induced phenotypic rescue and mitigated pathological progression in dystrophic mice without detectable toxicity. Improved membrane integrity conferred by exosomes inhibited intracellular calcium influx and calcium-dependent activation of calpain proteases, preventing the degradation of the destabilized dystrophin-associated protein complex. We show that exosomes, particularly myotube-derived exosomes, induced functional improvements and alleviated muscle deterioration by stabilizing damaged muscle membrane in dystrophic mice. Our findings suggest that exosomes may have therapeutic implications for DMD and other diseases with compromised membranes.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.