ArticleJournal of virology2000
Kinetics of recombinant adeno-associated virus-mediated gene transfer.
Article in Journal of virology, 2000. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
What it found
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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.
The trial behind it
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
16 citing papers in PubMed, 46 citations in OpenAlex.
- CD8+ T cell activation occurs 24 hours after AAV administration and is driven by muscle promoter specificity.JCI insight · 2026Article
- Reducing off-target expression of mRNA therapeutics and vaccines in the liver with microRNA binding sites.Molecular therapy. Methods & clinical development · 2025Article
- Temporal restriction of Cas9 expression improves CRISPR-mediated deletion efficacy and fidelity.Molecular therapy. Nucleic acids · 2024Article
- AAV9-mediated FIG4 delivery prolongs life span in Charcot-Marie-Tooth disease type 4J mouse model.The Journal of clinical investigation · 2021Article
- Rapid transgene expression in multiple precursor cell types of adult rat subventricular zone mediated by adeno-associated type 1 vectors.Human gene therapy · 2012Article
- Cardiac gene therapy with SERCA2a: from bench to bedside.Journal of molecular and cellular cardiology · 2011Review
- Gene delivery of sarcoplasmic reticulum calcium ATPase inhibits ventricular remodeling in ischemic mitral regurgitation.Circulation. Heart failure · 2010Article
- Cystic fibrosis: exploiting its genetic basis in the hunt for new therapies.Pharmacology & therapeutics · 2010Review
- Cardiac gene therapy.Seminars in thoracic and cardiovascular surgery · 2010Review
- Recombinant adeno-associated virus type 2-mediated gene delivery into theGenetic vaccines and therapy · 2004Article
- Immunity to adenovirus and adeno-associated viral vectors: implications for gene therapy.Gene therapy · 2003Review
- Genetic fate of recombinant adeno-associated virus vector genomes in muscle.Journal of virology · 2003Article
- Lymphangiogenic gene therapy with minimal blood vascular side effects.The Journal of experimental medicine · 2002Article
- TrkB gene transfer protects retinal ganglion cells from axotomy-induced death in vivo.The Journal of neuroscience : the official journal of the Society for Neuroscience · 2002Article
- Differential activation of innate immune responses by adenovirus and adeno-associated virus vectors.Journal of virology · 2002Article
- Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo.Journal of virology · 2001Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 3 institutions in 1 country.
Funding
Abstract
Recombinant adeno-associated virus (rAAV) vectors have been shown to be useful for efficient gene delivery to a variety of dividing and nondividing cells. Mechanisms responsible for the long-term, persistent expression of the rAAV transgene are not well understood. In this study we investigated the kinetics of rAAV-mediated human factor IX (hFIX) gene transfer into human primary myoblasts and myotubes. Transduction of both myoblasts and myotubes occured with a similar and high efficiency. After 3 to 4 weeks of transduction, rAAV with a cytomegalovirus (CMV) promoter showed 10- to 15-fold higher expression than that with a muscle-specific creatine kinase enhancer linked to beta-actin promoter. Factor IX expression from transduced myoblasts as well as myotubes reached levels as high as approximately 2 microgram of hFIX/10(6) cells/day. Southern blot analyses of high-molecular-weight (HMW) cellular genomic and Hirt DNAs isolated from rAAV/CMVhFIXm1-transduced cells showed that the conversion of single-stranded vector genomes to double-stranded DNA forms, but not the level of the integrated forms in HMW DNA, correlated with increasing expression of the transgene. Together, these results indicate that rAAV can transduce both proliferating and terminally differentiated muscle cells at about the same efficiency, that expression of transgenes increases linearly over their lifetime with no initial lag phase, and that increasing expression correlates with the appearance of double-stranded episomal rAAV genomes. Evidence showing that the rAAV virions can copackage hFIX, presumably nonspecifically, was also obtained.
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What OpenQuestion holds
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.