ArticleGene therapy2001
Transfection of large plasmids in primary human myoblasts.
Article in Gene therapy, 2001. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
25 citing papers in PubMed, 86 citations in OpenAlex.
- Evaluation of DNA minicircles for delivery of adenine and cytosine base editors using activatable gene on "GO" reporter imaging systems.Molecular therapy. Nucleic acids · 2024Article
- Non-viral delivery of nucleic acid for treatment of rare diseases of the muscle.Journal of biosciences · 2024Review
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- Poly (β-amino Ester) Nanoparticles Modified with a Rabies Virus-derived peptide for the Delivery ofACS applied nano materials · 2023Article
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- Production and Use of Gesicles for Nucleic Acid Delivery.Molecular biotechnology · 2022Article
- Development of Minicircle Vectors Encoding COL7A1 Gene with Human Promoters for Non-Viral Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa.International journal of molecular sciences · 2021Article
- Genetic code expansion in mammalian cells: A plasmid system comparison.Bioorganic & medicinal chemistry · 2020Article
- Efficient Transfection of Large Plasmids Encoding HIV-1 into Human Cells-A High Potential Transfection System Based on a Peptide Mimicking Cationic Lipid.Pharmaceutics · 2020Article
- Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Therapeutics.The AAPS journal · 2018Review
- Synthetically controlling dendrimer flexibility improves delivery of large plasmid DNA.Chemical science · 2017Article
- Minicircle DNA Provides Enhanced and Prolonged Transgene Expression Following Airway Gene Transfer.Scientific reports · 2016Article
- Comparison of Different Electroporation Parameters on Transfection Efficiency of Sheep Testicular Cells.Cell journal · 2016Article
- Article
- In vitro assessments of nanomaterial toxicity.Advanced drug delivery reviews · 2009Review
- Drug releasing systems in cardiovascular tissue engineering.Journal of cellular and molecular medicine · 2009Review
- Mini-dystrophin efficiently incorporates into the dystrophin protein complex in living cells.Journal of muscle research and cell motility · 2006Article
- Nonviral gene transfer to skeletal, smooth, and cardiac muscle in living animals.American journal of physiology. Cell physiology · 2005Review
- Polymers for DNA delivery.Molecules (Basel, Switzerland) · 2005Review
- Muscle stem cells can act as antigen-presenting cells: implication for gene therapy.Gene therapy · 2004Article
Corrections and comments
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Authors and funding
5 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The ex vivo gene therapy approach for Duchenne muscular dystrophy is promising since myoblast transplantation in primates is now very efficient. One obstacle to this treatment is the low transfection efficiency of large DNA constructs in human primary myoblasts. Small plasmids can be easily transfected with the new phosphonolipid described in this study. However, a dramatic drop in transfection efficiency is observed with plasmids of 12 kb or more containing EGFP minidystrophin and EGFP dystrophin fusion genes. The transfection of human primary myoblasts with such large plasmids could only be achieved when the DNA was linked to an adenovirus with the use of polyethylenimine (PEI), with efficiencies ranging between 3 and 5% of transitory transfection. Branched 2 kDa PEI was less toxic in PEI adenofection than branched 25 kDa PEI or linear 22 kDa PEI. The adenovirus was an absolute necessity for an efficient transfection. An integrin-binding peptide, a nuclear localization signal peptide, chloroquine, glycerol or cell cycle synchronization using aphidicolin did not enhance PEI adenofection. Following PEI adenofection, the adenoviral proteins were detected using a polyclonal antibody. The detected antigens fell below the detectable level after 12 days in culture. We thus provide in this study an efficient and reproducible method to permit efficient delivery of large plasmids to human primary myoblasts for the ex vivo gene therapy of Duchenne muscular dystrophy.
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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.