ArticleSkeletal muscle2015
Differential myofiber-type transduction preference of adeno-associated virus serotypes 6 and 9.
Article in Skeletal muscle, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.
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Who cites it
23 citing papers in PubMed.
- Preclinical efficacy of a gene therapy forMolecular therapy. Advances · 2026Article
- Codon-optimized human Smad7 gene therapy enhances skeletal muscle mass and function in a murine model of Duchenne muscular dystrophy.Gene therapy · 2026Article
- Adeno-Associated Virus 8 and 9 Myofibre Type/Size Tropism Profiling Reveals Therapeutic Effect of Microdystrophin in Canines.Journal of cachexia, sarcopenia and muscle · 2025Article
- Inhibition of miR-25 ameliorates cardiac and skeletal muscle dysfunction in agedMolecular therapy. Nucleic acids · 2024Article
- Comparative in vivo characterization of newly discovered myotropic adeno-associated vectors.Skeletal muscle · 2024Article
- Chronic aryl hydrocarbon receptor activity impairs muscle mitochondrial function with tobacco smoking.Journal of cachexia, sarcopenia and muscle · 2024Article
- Local and systemic administration of AAV vectors with alphaherpesvirus latency-associated promoter 2 drives potent transgene expression in mouse liver, kidney, and skeletal muscle.Journal of virological methods · 2023Article
- Codon-optimized FAM132b gene therapy prevents dietary obesity by blockading adrenergic response and insulin action.International journal of obesity (2005) · 2022Article
- Optogenetic activation of the diaphragm.Scientific reports · 2022Article
- Discovering fiber type architecture over the entire muscle using data-driven analysis.Cytometry. Part A : the journal of the International Society for Analytical Cytology · 2021Article
- FGF-2-dependent signaling activated in aged human skeletal muscle promotes intramuscular adipogenesis.Proceedings of the National Academy of Sciences of the United States of America · 2021Article
- AUF1 gene transfer increases exercise performance and improves skeletal muscle deficit in adult mice.Molecular therapy. Methods & clinical development · 2021Article
- Of rAAV and Men: From Genetic Neuromuscular Disorder Efficacy and Toxicity Preclinical Studies to Clinical Trials and Back.Journal of personalized medicine · 2020Review
- Cytoskeletal disorganization underlies PABPN1-mediated myogenic disability.Scientific reports · 2020Article
- Transduction Efficiency of Adeno-Associated Virus Serotypes After Local Injection in Mouse and Human Skeletal Muscle.Human gene therapy · 2020Article
- An Engineered AAV6-Based Vaccine Induces High Cytolytic Anti-Tumor Activity by Directly Targeting DCs and Improves Ag Presentation.Molecular therapy oncolytics · 2019Article
- A Novel AAV-mediated Gene Delivery System Corrects CFTR Function in Pigs.American journal of respiratory cell and molecular biology · 2019Article
- Mitochondrial glycerol 3-phosphate dehydrogenase promotes skeletal muscle regeneration.EMBO molecular medicine · 2018Article
- Proteasomal activity-based probes mark protein homeostasis in muscles.Journal of cachexia, sarcopenia and muscle · 2017Article
- A compact unc45b-promoter drives muscle-specific expression in zebrafish and mouse.Genesis (New York, N.Y. : 2000) · 2016Article
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Authors and funding
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundGene therapy strategies are promising therapeutic options for monogenic muscular dystrophies, with several currently underways. The adeno-associated viral (AAV) vector is among the most effective gene delivery systems. However, transduction efficiency in skeletal muscles varies between AAV serotypes, with the underlying factors poorly understood. We hypothesized that myofiber-specific tropism differs between AAV serotypes.
methodsWe developed a quantitative histology procedure and generated myofiber pattern maps for four myosin heavy chain (MyHC) isotypes. We compared myofiber pattern maps between AAV6 or AAV9 injected tibialis anterior muscle in mice. We correlated MyHC expression with AAV-derived green fluorescence protein (GFP) expression using statistical models.
resultsWe found that MyHC-2x expressing myofibers display a significantly higher preference for AAV transduction, whereas MyHC-2b expressing myofibers negatively correlated with AAV transduction. In addition, we show that AAV9-mediated transduction is enriched in myofibers expressing MyHC-1 and MyHC-1/2a. Moreover, AAV9-mediated transduction can predominantly be predicted by the expression of MyHC isotypes. In contrast, AAV6 transduction can be predicted by myofiber size but not by myofiber types.
conclusionsOur findings identify differences between AAV6 and AAV9 for myofiber-type preferences, which could be an underlying factor for mosaic transduction of skeletal muscle. Adjusting AAV serotype for specific muscle conditions can therefore improve transduction efficacy in clinical applications.
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