ArticleMolecular therapy : the journal of the American Society of Gene Therapy2015
Hybrid nonviral/viral vector systems for improved piggyBac DNA transposon in vivo delivery.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers.
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Who cites it
29 citing papers in PubMed, 46 citations in OpenAlex.
- Gene therapy for liver diseases: methods, challenges and opportunities.Journal of nanobiotechnology · 2026Review
- Inhalable gene and RNA therapy for cystic fibrosis: perspectives and progress in clinical development.Nanomedicine (London, England) · 2026Review
- Recent Advances in Therapeutics and Manufacturing Processes of Recombinant Adeno-Associated Virus for the Treatment of Lung Diseases.Current gene therapy · 2025Review
- Evaluation of anti-vector immune responses to adenovirus-mediated lung gene therapy and modulation by αCD20.Molecular therapy. Methods & clinical development · 2024Article
- High ionic strength vector formulations enhance gene transfer to airway epithelia.Nucleic acids research · 2024Article
- tRNA therapeutics for genetic diseases.Nature reviews. Drug discovery · 2024Review
- High ionic strength vector formulations enhance gene transfer to airway epithelia.bioRxiv : the preprint server for biology · 2024Article
- Article
- Application and Design of Switches Used in CAR.Cells · 2022Review
- Tissue and cell-type-specific transduction using rAAV vectors in lung diseases.Journal of molecular medicine (Berlin, Germany) · 2021Review
- Gene Therapy Potential for Genetic Disorders of Surfactant Dysfunction.Frontiers in genome editing · 2021Review
- Treatment of Cystic Fibrosis: From Gene- to Cell-Based Therapies.Frontiers in pharmacology · 2021Review
- Adeno-Associated Virus-Based Gene Therapy for Lifelong Correction of Genetic Disease.Human gene therapy · 2020Review
- A viral toolkit for recording transcription factor-DNA interactions in live mouse tissues.Proceedings of the National Academy of Sciences of the United States of America · 2020Article
- Article
- Review
- A Novel AAV-mediated Gene Delivery System Corrects CFTR Function in Pigs.American journal of respiratory cell and molecular biology · 2019Article
- Enhanced Tropism of Species B1 Adenoviral-Based Vectors for Primary Human Airway Epithelial Cells.Molecular therapy. Methods & clinical development · 2019Article
- In VivoInternational journal of molecular sciences · 2019Article
- Self-assembled peptide-poloxamine nanoparticles enable in vitro and in vivo genome restoration for cystic fibrosis.Nature nanotechnology · 2019Article
Corrections and comments
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Authors and funding
3 authors at 1 institution in 1 country.
Funding
Abstract
The DNA transposon piggyBac is a potential therapeutic agent for multiple genetic diseases such as cystic fibrosis (CF). Recombinant piggyBac transposon and transposase are typically codelivered by plasmid transfection; however, plasmid delivery is inefficient in somatic cells in vivo and is a barrier to the therapeutic application of transposon-based vector systems. Here, we investigate the potential for hybrid piggyBac/viral vectors to transduce cells and support transposase-mediated genomic integration of the transposon. We tested both adenovirus (Ad) and adeno-associated virus (AAV) as transposon delivery vehicles. An Ad vector expressing hyperactive insect piggyBac transposase (iPB7) was codelivered. We show transposase-dependent transposition activity and mapped integrations in mammalian cells in vitro and in vivo from each viral vector platform. We also demonstrate efficient and persistent transgene expression following nasal delivery of piggyBac/viral vectors to mice. Furthermore, using piggyBac/Ad expressing Cystic Fibrosis transmembrane Conductance Regulator (CFTR), we show persistent correction of chloride current in well-differentiated primary cultures of human airway epithelial cells derived from CF patients. Combining the emerging technologies of DNA transposon-based vectors with well-studied adenoviral and AAV delivery provides new tools for in vivo gene transfer and presents an exciting opportunity to increase the delivery efficiency for therapeutic genes such as CFTR.
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Registered trials
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.