ArticleNucleic acids research2013
A concept of eliminating nonhomologous recombination for scalable and safe AAV vector generation for human gene therapy.
Article in Nucleic acids research, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
22 citing papers in PubMed.
- An Improved Method for Determining the Infection Titer of Replication-Competent Adeno-Associated Virus.Biomedicines · 2026Article
- Reduced rAAV interference enhances rcAAV detection sensitivity.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
- AAVone: A cost-effective, single-plasmid solution for efficient AAV production with reduced DNA impurities.Molecular therapy. Nucleic acids · 2025Article
- Thorough molecular configuration analysis of noncanonical AAV genomes in AAV vector preparations.Molecular therapy. Methods & clinical development · 2024Article
- Long-Term Metabolic Correction of Phenylketonuria by AAV-Delivered Phenylalanine Amino Lyase.Molecular therapy. Methods & clinical development · 2020Article
- Nanopore sequencing of native adeno-associated virus (AAV) single-stranded DNA using a transposase-based rapid protocol.NAR genomics and bioinformatics · 2020Article
- Enhanced Transduction of Human Hematopoietic Stem Cells by AAV6 Vectors: Implications in Gene Therapy and Genome Editing.Molecular therapy. Nucleic acids · 2020Article
- Internal Ribosome Entry Site Dramatically Reduces Transgene Expression in Hematopoietic Cells in a Position-Dependent Manner.Viruses · 2019Article
- Pharmacology of Recombinant Adeno-associated Virus Production.Molecular therapy. Methods & clinical development · 2018Review
- Molecular design for recombinant adeno-associated virus (rAAV) vector production.Applied microbiology and biotechnology · 2018Review
- A Robust System for Production of Superabundant VP1 Recombinant AAV Vectors.Molecular therapy. Methods & clinical development · 2017Article
- The hepatocyte-specific HNF4α/miR-122 pathway contributes to iron overload-mediated hepatic inflammation.Blood · 2017Article
- Syngeneic AAV Pseudo-particles Potentiate Gene Transduction of AAV Vectors.Molecular therapy. Methods & clinical development · 2017Article
- High-Density Recombinant Adeno-Associated Viral Particles are Competent Vectors for In Vivo Transduction.Human gene therapy · 2016Article
- High-Efficiency Transduction of Primary Human Hematopoietic Stem/Progenitor Cells by AAV6 Vectors: Strategies for Overcoming Donor-Variation and Implications in Genome Editing.Scientific reports · 2016Article
- Development of Optimized AAV Serotype Vectors for High-Efficiency Transduction at Further Reduced Doses.Human gene therapy methods · 2016Article
- Strategies to generate high-titer, high-potency recombinant AAV3 serotype vectors.Molecular therapy. Methods & clinical development · 2016Article
- Scalable downstream strategies for purification of recombinant adeno- associated virus vectors in light of the properties.Current pharmaceutical biotechnology · 2015Review
- Vaccinia virus as a subhelper for AAV replication and packaging.Molecular therapy. Methods & clinical development · 2015Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors.
Funding
Abstract
Scalable and efficient production of high-quality recombinant adeno-associated virus (rAAV) for gene therapy remains a challenge despite recent clinical successes. We developed a new strategy for scalable and efficient rAAV production by sequestering the AAV helper genes and the rAAV vector DNA in two different subcellular compartments, made possible by using cytoplasmic vaccinia virus as a carrier for the AAV helper genes. For the first time, the contamination of replication-competent AAV particles (rcAAV) can be completely eliminated in theory by avoiding ubiquitous nonhomologous recombination. Vector DNA can be integrated into the host genomes or delivered by a nuclear targeting vector such as adenovirus. In suspension HeLa cells, the achieved vector yield per cell is similar to that from traditional triple-plasmid transfection method. The rcAAV contamination was undetectable at the limit of our assay. Furthermore, this new concept can be used not only for production of rAAV, but also for other DNA vectors.
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.