ArticlePloS one2014
Delivery of full-length factor VIII using a piggyBac transposon vector to correct a mouse model of hemophilia A.
Article in PloS one, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 33 papers.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
33 citing papers in PubMed.
- Deconstructing gene therapy in hemophilia for the clinician.Hematology. American Society of Hematology. Education Program · 2025Review
- CCL5 paradoxically regulates glomerular injury by skewing macrophage polarization.JCI insight · 2025Article
- Transformative approaches in hemophilia management: from traditional therapies to prenatal stem cell treatment.Frontiers in bioengineering and biotechnology · 2025Review
- Non-viral and viral delivery systems for hemophilia A therapy: recent development and prospects.Annals of hematology · 2024Review
- Applications and Research Advances in the Delivery of CRISPR/Cas9 Systems for the Treatment of Inherited Diseases.International journal of molecular sciences · 2023Review
- Impaired factor V-related anticoagulant mechanisms and deep vein thrombosis associated with A2086D and W1920R mutations.Blood advances · 2023Article
- Localized astrogenesis regulates gyrification of the cerebral cortex.Science advances · 2022Article
- Transplantation of human iPSC-derived muscle stem cells in the diaphragm of Duchenne muscular dystrophy model mice.PloS one · 2022Article
- Development of alternative gene transfer techniques forRegenerative therapy · 2021Article
- Potential of Induced Pluripotent Stem Cells for Use in Gene Therapy: History, Molecular Bases, and Medical Perspectives.Biomolecules · 2021Review
- Utilization of a novel Sendai virus vector in ex vivo gene therapy for hemophilia A.International journal of hematology · 2021Article
- Glial cell type-specific gene expression in the mouse cerebrum using the piggyBac system and in utero electroporation.Scientific reports · 2021Article
- CRISPR/Cas System and Factors Affecting Its Precision and Efficiency.Frontiers in cell and developmental biology · 2021Review
- A non-viral genome editing platform for site-specific insertion of large transgenes.Stem cell research & therapy · 2020Article
- Review
- BioID screening of biotinylation sites using the avidin-like protein Tamavidin 2-REV identifies global interactors of stimulator of interferon genes (STING).The Journal of biological chemistry · 2020Article
- Review
- Advances in gene therapy for hemophilia.Journal of biosciences · 2020Review
- Advances in gene therapy for hemophilia: basis, current status, and future perspectives.International journal of hematology · 2020Review
- Bioengineering hemophilia A-specific microvascular grafts for delivery of full-length factor VIII into the bloodstream.Blood advances · 2019Article
Corrections and comments
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Authors and funding
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Viral vectors have been used for hemophilia A gene therapy. However, due to its large size, full-length Factor VIII (FVIII) cDNA has not been successfully delivered using conventional viral vectors. Moreover, viral vectors may pose safety risks, e.g., adverse immunological reactions or virus-mediated cytotoxicity. Here, we took advantages of the non-viral vector gene delivery system based on piggyBac DNA transposon to transfer the full-length FVIII cDNA, for the purpose of treating hemophilia A. We tested the efficiency of this new vector system in human 293T cells and iPS cells, and confirmed the expression of the full-length FVIII in culture media using activity-sensitive coagulation assays. Hydrodynamic injection of the piggyBac vectors into hemophilia A mice temporally treated with an immunosuppressant resulted in stable production of circulating FVIII for over 300 days without development of anti-FVIII antibodies. Furthermore, tail-clip assay revealed significant improvement of blood coagulation time in the treated mice. piggyBac transposon vectors can facilitate the long-term expression of therapeutic transgenes in vitro and in vivo. This novel gene transfer strategy should provide safe and efficient delivery of FVIII.
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