ArticleProceedings of the National Academy of Sciences of the United States of America2005
Correction of chromosomal mutation and random integration in embryonic stem cells with helper-dependent adenoviral vectors.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2005. 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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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.
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Who cites it
29 citing papers in PubMed, 61 citations in OpenAlex.
- Optimizing Lentiviral Vector Production: Insights Into PiggyBac Transposase and Concatemeric Array Strategies.Biotechnology journal · 2025Article
- A Single "All-in-One" Helper-Dependent Adenovirus to Deliver Donor DNA and CRISPR/Cas9 for Efficient Homology-Directed Repair.Molecular therapy. Methods & clinical development · 2020Article
- High-Capacity Adenoviral Vectors: Expanding the Scope of Gene Therapy.International journal of molecular sciences · 2020Review
- Review
- Bi-allelic Homology-Directed Repair with Helper-Dependent Adenoviruses.Molecular therapy. Methods & clinical development · 2019Article
- Targeted Integration and High-Level Transgene Expression in AAVS1 Transgenic Mice after In Vivo HSC Transduction with HDAd5/35++ Vectors.Molecular therapy : the journal of the American Society of Gene Therapy · 2019Article
- Adenovirus vectors in hematopoietic stem cell genome editing.FEBS letters · 2019Review
- Production of CRISPR/Cas9-Mediated Self-Cleaving Helper-Dependent Adenoviruses.Molecular therapy. Methods & clinical development · 2019Article
- Transcriptional profiling of isogenic Friedreich ataxia neurons and effect of an HDAC inhibitor on disease signatures.The Journal of biological chemistry · 2019Article
- Assessing the Effectiveness of a Far-Red Fluorescent Reporter for Tracking Stem Cells In Vivo.International journal of molecular sciences · 2017Article
- Gene Editing with Helper-Dependent Adenovirus Can Efficiently Introduce Multiple Changes Simultaneously over a Large Genomic Region.Molecular therapy. Nucleic acids · 2017Article
- Homology Requirements for Efficient, Footprintless Gene Editing at the CFTR Locus in Human iPSCs with Helper-dependent Adenoviral Vectors.Molecular therapy. Nucleic acids · 2016Article
- Engineered Viruses as Genome Editing Devices.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Review
- BTK gene targeting by homologous recombination using a helper-dependent adenovirus/adeno-associated virus hybrid vector.Gene therapy · 2016Article
- Helper-dependent adenoviral vectors for liver-directed gene therapy of primary hyperoxaluria type 1.Gene therapy · 2016Article
- Efficient and accurate homologous recombination in hESCs and hiPSCs using helper-dependent adenoviral vectors.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Helper-Dependent Adenoviral Vectors.Journal of genetic syndromes & gene therapy · 2011Article
- Article
- Genomic integration of adenoviral gene transfer vectors following transduction of fertilized mouse oocytes.Transgenic research · 2011Article
- Hyperactive sleeping beauty transposase enables persistent phenotypic correction in mice and a canine model for hemophilia B.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Article
Corrections and comments
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Authors and funding
9 authors at 1 institution in 1 country.
Funding
Abstract
For gene therapy of inherited diseases, targeted integration/gene repair through homologous recombination (HR) between exogenous and chromosomal DNA would be an ideal strategy to avoid potentially serious problems of random integration such as cellular transformation and gene silencing. Efficient sequence-specific modification of chromosomes by HR would also advance both biological studies and therapeutic applications of a variety of stem cells. Toward these goals, we developed an improved strategy of adenoviral vector (AdV)-mediated HR and examined its ability to correct an insertional mutation in the hypoxanthine phosphoribosyl transferase (Hprt) locus in male mouse ES cells. The efficiency of HR was compared between four types of AdVs that contained various lengths of homologies at the Hprt locus and with various multiplicities of infections. The frequency of HR with helper-dependent AdVs (HD AdVs) with an 18.6-kb homology reached 0.2% per transduced cell at a multiplicity of infection of 10 genomes per cell. Detection of random integration at DNA levels by PCR revealed extremely high efficiency of 5% per cell. We also isolated and characterized chromosomal sites where HD AdVs integrated in a random manner. In contrast to retroviral, lentiviral, and adeno-associated viral vectors, which tend to integrate into genes, the integration sites of AdV was distributed randomly inside and outside genes. These findings suggest that HR mediated by HD AdVs is efficient and relatively safe and might be a new viable option for ex vivo gene therapy as well as a tool for chromosomal manipulation of a variety of stem cells.
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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.