Single-Walled carbon nanotubes for precision treatment of Duchenne muscular dystrophy: a mini review

Duchenne Muscular Dystrophy (DMD) is a severe X-linked neuromuscular disorder characterized by progressive muscle degeneration due to mutations in the dystrophin gene. This review aims to critically assess the application of Single-Walled Carbon Nanotubes (SWCNTs) as advanced nanocarriers for DMD treatment. It focuses on overcoming limitations of current strategies—such as poor bioavailability, low targeting efficiency, and off-target toxicity—by leveraging the physicochemical versatility and functionalization potential of SWCNTs.Single-Walled Carbon Nanotubes (SWCNTs) have emerged as a promising nanocarrier system for precision treatment of DMD, offering superior drug-loading capacity, targeted delivery, and enhanced cellular uptake.Their high surface area (~۱۳۱۵ m²/g) and tunable functionalization enable efficient transport of antisense oligonucleotides (ASOs), phosphorodiamidate morpholino oligomers (PMOs), and CRISPR/Cas۹ gene-editing complexes to dystrophic muscle fibers. Preclinical studies indicate ۷۰% exon-skipping efficiency and ۵۵% dystrophin restoration with SWCNT-based PMOs, alongside ۸-fold higher genome correction efficiency in CRISPR applications. Additionally, SWCNTs exhibit prolonged circulation, improved muscle tissue penetration, and reduced off-target accumulation compared to lipid nanoparticles (LNPs). However, safety concerns such as potential oxidative stress, immune interactions, and long-term biodegradability remain key challenges for clinical translation. Functionalization strategies, AI-driven molecular modeling, and targeted clearance mechanisms are being explored to optimize SWCNT biocompatibility.By addressing current translational barriers—including toxicity, immunogenicity, and large-scale production—SWCNT-based platforms hold substantial promise as next-generation precision therapies for DMD. Their integration into personalized nanomedicine frameworks could redefine treatment paradigms in neuromuscular disorders. Addressing current limitations will be crucial in harnessing SWCNTs as a next-generation precision therapy for DMD, paving the way for personalized nanomedicine applications in neuromuscular disorders.

کلیدواژه ها:

Duchenne muscular dystrophy ، Single-Walled carbon nanotubes ، Exon-skipping ، Gene Therapy ، Drug Delivery ، Nanomedicine