Introduction: Muscular dystrophies (MDs) are inherited neuromuscular disorders characterized by progressive muscle degeneration and functional decline. Conventional therapies such as corticosteroids and physiotherapy offer only symptomatic relief, highlighting the need for molecularly targeted approaches. Nanomedicine provides new opportunities for precise drug and gene delivery to dystrophic muscle tissues. Methods: Recent peer-reviewed studies (2020-2024) were identified through PubMed, Scopus, and Web of Science using the keywords “nanomedicine,” “nanoparticles,” “muscular dystrophy,” and “gene therapy.” Articles addressing therapeutic mechanisms, delivery efficiency, and translational outcomes were systematically analyzed. Results: Key nanocarriers such as liposomes, PLGA nanoparticles, and exosomes exhibit distinct mechanisms for overcoming muscle delivery barriers. Liposomes improve membrane fusion and drug solubility but suffer from low stability and rapid clearance. PLGA nanoparticles enable sustained release and controlled biodistribution, though polymer degradation may induce local acidity. Exosomes, as endogenous vesicles, show superior biocompatibility, low immunogenicity, and natural muscle tropism, effectively penetrating fibrotic extracellular matrices. Preclinical data demonstrate enhanced dystrophin restoration and muscle function using nanoparticle-mediated CRISPR/Cas9 systems. Discussion: These findings underscore the importance of optimizing nanocarrier design to balance delivery efficiency, biosafety, and scalability. Exosome-based vectors, in particular, address major translational barriers such as immune activation and off-target toxicity, positioning them as promising next-generation carriers. Conclusion: Nanomedicine offers a transformative and biocompatible platform for safe, targeted, and durable genetic correction in MDs, bridging the gap between preclinical innovation and clinical translation.
Manne et al. (Mon,) studied this question.