ArticleNucleic acids research2016
piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts.
Article in Nucleic acids research, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
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Who cites it
17 citing papers in PubMed, 26 citations in OpenAlex.
- CARAMBA: a first-in-human clinical trial with SLAMF7 CAR-T cells prepared by virus-free Sleeping Beauty gene transfer to treat multiple myeloma.Gene therapy · 2021Trial
- Local Non-Coding Regulatory Elements in Muscular Dystrophies.International journal of molecular sciences · 2025Review
- Challenges and Considerations of Preclinical Development for iPSC-Based Myogenic Cell Therapy.Cells · 2024Review
- Find and cut-and-transfer (FiCAT) mammalian genome engineering.Nature communications · 2021Article
- Applications ofStem cells international · 2021Review
- Review
- Jump around: transposons in and out of the laboratory.F1000Research · 2020Review
- The effect of type 2 diabetes mellitus and obesity on muscle progenitor cell function.Stem cell research & therapy · 2019Review
- The impact of transposable element activity on therapeutically relevant human stem cells.Mobile DNA · 2019Review
- The mesenchymoangioblast, mesodermal precursor for mesenchymal and endothelial cells.Cellular and molecular life sciences : CMLS · 2018Review
- Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells.Nucleic acids research · 2018Article
- Autologous Cell Therapy Approach for Duchenne Muscular Dystrophy using PiggyBac Transposons and Mesoangioblasts.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Article
- Combined Therapies for Duchenne Muscular Dystrophy to Optimize Treatment Efficacy.Frontiers in genetics · 2018Review
- Preclinical and clinical advances in transposon-based gene therapy.Bioscience reports · 2017Review
- The golden retriever model of Duchenne muscular dystrophy.Skeletal muscle · 2017Review
- Endogenous Transposase Source in Human Cells Mobilizes piggyBac Transposons.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Article
- CRISPR/Cas9 Flexes Its Muscles: In Vivo Somatic Gene Editing for Muscular Dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Article
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Authors and funding
14 authors at 4 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Duchenne muscular dystrophy (DMD) is a genetic neuromuscular disorder caused by the absence of dystrophin. We developed a novel gene therapy approach based on the use of the piggyBac (PB) transposon system to deliver the coding DNA sequence (CDS) of either full-length human dystrophin (DYS: 11.1 kb) or truncated microdystrophins (MD1: 3.6 kb; MD2: 4 kb). PB transposons encoding microdystrophins were transfected in C2C12 myoblasts, yielding 65±2% MD1 and 66±2% MD2 expression in differentiated multinucleated myotubes. A hyperactive PB (hyPB) transposase was then deployed to enable transposition of the large-size PB transposon (17 kb) encoding the full-length DYS and green fluorescence protein (GFP). Stable GFP expression attaining 78±3% could be achieved in the C2C12 myoblasts that had undergone transposition. Western blot analysis demonstrated expression of the full-length human DYS protein in myotubes. Subsequently, dystrophic mesoangioblasts from a Golden Retriever muscular dystrophy dog were transfected with the large-size PB transposon resulting in 50±5% GFP-expressing cells after stable transposition. This was consistent with correction of the differentiated dystrophic mesoangioblasts following expression of full-length human DYS. These results pave the way toward a novel non-viral gene therapy approach for DMD using PB transposons underscoring their potential to deliver large therapeutic genes.
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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.