ArticleMolecular therapy. Methods & clinical development2025
Systemic delivery of AAV5, AAV8, and AAV9 packaging a C5-12-microdystrophin-FLAG expression cassette in non-human primates.
Article in Molecular therapy. Methods & clinical development, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.
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Who cites it
5 citing papers in PubMed.
- Dose- and genotype-dependent cardiac arrhythmia and sudden death in rats following microdystrophin gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- From molecular correction to functional rescue: delivery, tissue state, and immunobiology as the principal constraints on dystrophin-restoring therapy in Duchenne muscular dystrophy.Frontiers in pharmacology · 2026Review
- Gene replacement therapy for Piga GPI-anchor deficiency in the developing nervous system.bioRxiv : the preprint server for biology · 2025Article
- Engineering Targeted Gene Delivery Systems for Primary Hereditary Skeletal Myopathies: Current Strategies and Future Perspectives.Biomedicines · 2025Review
- Advancing adeno-associated virus for Duchenne muscular dystrophy treatment: Moving beyond rodent models.Molecular therapy. Methods & clinical development · 2025Article
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Authors and funding
14 authors.
Funding
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Abstract
Safely achieving therapeutic expression levels with adeno-associated virus (AAV) gene therapy is a significant challenge for treating the large muscle mass in humans. Non-human primates (NHPs) provide a more accurate assessment of the feasibility of achieving an effective and safe dose than rodents. Here, we compared a single systemic administration of AAV5, AAV8, or AAV9 in NHPs, each packaging the C5-12-microdystrophin-FLAG expression cassette. At 1 month post-dose, we compared tissue vector genomes, mRNA, and microdystrophin-FLAG protein levels by meso-scale discovery-enzyme-linked immunosorbent assay, liquid chromatography-mass spectrometry, and immunofluorescence. The C5-12 promoter was highly selective for heart and skeletal muscles, when compared to off-target tissues such as peripheral blood mononuclear cells, lung, liver, and kidney. AAV8 led to higher levels of microdystrophin-FLAG mRNA and protein in the cardiac ventricles and skeletal muscles when compared to AAV5 or AAV9. The AAV8-microdystrophin-FLAG led to ∼20% of wild-type NHP dystrophin protein expression levels and was located on the sarcolemma of ∼40% of skeletal muscles fibers and ∼15% of left ventricular cardiomyocytes. Hematology, serum chemistry, and pathology were unremarkable. Thus, a systemic dose of ∼1.18 × 10
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