ArticleGene therapy2002
Replication competent helper functions for recombinant AAV vector generation.
Article in Gene therapy, 2002. 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, 23 citations in OpenAlex.
- Combined Plasmid Redesign and Transfection Optimization Significantly Increases Upstream AAV Titers While Maintaining Vector Quality and In Vivo Potency.Microorganisms · 2026Article
- Backbone-Minimised Nanoplasmid DNA Systems Enable High-Titre AAV Production in Suspension HEK293 Platforms.Pharmaceutics · 2026Article
- Chronologically distributed transfection improves AAV2 and AAV2/8 capsid filling and reveals assembly schedule divergence.Molecular therapy. Methods & clinical development · 2025Article
- Development of cell-based assay for detecting replication-competent adeno-associated virus by qPCR.Molecular therapy. Methods & clinical development · 2025Article
- Inducible HEK293 AAV packaging cell lines expressing Rep proteins.Molecular therapy. Methods & clinical development · 2023Article
- Gene-encoding DNA origami for mammalian cell expression.Nature communications · 2023Article
- Enhanced sensitivity of neutralizing antibody detection for different AAV serotypes using HeLa cells with overexpressed AAVR.Frontiers in pharmacology · 2023Article
- Viral Vector Systems for Gene Therapy: A Comprehensive Literature Review of Progress and Biosafety Challenges.Applied biosafety : journal of the American Biological Safety Association · 2020Review
- Developing an Anion Exchange Chromatography Assay for Determining Empty and Full Capsid Contents in AAV6.2.Molecular therapy. Methods & clinical development · 2019Article
- AAV9 Vector: a Novel modality in gene therapy for spinal muscular atrophy.Gene therapy · 2019Review
- Development of a Novel Recombinant Adeno-Associated Virus Production System Using Human Bocavirus 1 Helper Genes.Molecular therapy. Methods & clinical development · 2018Article
- Systematic Comparison and Validation of Quantitative Real-Time PCR Methods for the Quantitation of Adeno-Associated Viral Products.Human gene therapy methods · 2015Article
- Vaccinia virus as a subhelper for AAV replication and packaging.Molecular therapy. Methods & clinical development · 2015Article
- Gene delivery with viral vectors for cerebrovascular diseases.Frontiers in bioscience (Elite edition) · 2013Review
- Characterization of genome integrity for oversized recombinant AAV vector.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Article
- Improved splicing of adeno-associated viral (AAV) capsid protein-supplying pre-mRNAs leads to increased recombinant AAV vector production.Human gene therapy · 2008Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 2 institutions in 1 country.
Funding
Abstract
Adeno-associated virus (AAV) is a promising gene transfer vector tested in both animal studies and human clinical trials. However, current production methods are generally inefficient and require improvements to meet the increasing clinical need for economical, high titer and high quality rAAV vectors. The inefficiency of the current systems largely arises from the AAV helper function, which contains only the AAV coding region but lacks inverted terminal repeats. The terminal repeats were originally removed to prevent replication competent AAV contamination. Here we designed a novel and highly efficient rAAV helper function containing AAV terminal repeats. The new helper function not only mimics the wild-type AAV growth as it replicates along with the vector plasmid, but also restores the cis regulating function of the AAV terminal repeats. Addition of heterologous introns to the helper genome and use of a mutant AAV terminal repeat defective in packaging effectively controls the contamination of replication competent AAV particles. This new strategy also performs better in AAV producing cell lines than those based on non-replicating AAV rep and cap genome.
Indexed as
Identifiers
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.