ArticleGene therapy2003
Targeted transgene integration into transgenic mouse fibroblasts carrying the full-length human AAVS1 locus mediated by HSV/AAV rep(+) hybrid amplicon vector.
Article in Gene therapy, 2003. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 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
16 citing papers in PubMed, 38 citations in OpenAlex.
- 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
- An HBV-tolerant immunocompetent model that effectively simulates chronic hepatitis B virus infection in mice.Experimental animals · 2016Article
- 4-Phenylbutyrate attenuates the ER stress response and cyclic AMP accumulation in DYT1 dystonia cell models.PloS one · 2014Article
- Imaging gene delivery in a mouse model of congenital neuronal ceroid lipofuscinosis.Gene therapy · 2011Article
- Virion-associated cofactor high-mobility group DNA-binding protein-1 facilitates transposition from the herpes simplex virus/Sleeping Beauty amplicon vector platform.Human gene therapy · 2010Article
- Adeno-associated virus: a key to the human genome?Future virology · 2010Article
- Herpes simplex virus type 1/adeno-associated virus hybrid vectors.The open virology journal · 2010Article
- A 16-bp RBE element mediated Rep-dependent site-specific integration in AAVS1 transgenic mice for expression of hFIX.Gene therapy · 2009Article
- Preferential integration of adeno-associated virus type 2 into a polypyrimidine/polypurine-rich region within AAVS1.Journal of virology · 2007Article
- Improvement in reproductive parameters in hypogonadal female mice by regulated gene replacement therapy in the central nervous system.Gene therapy · 2007Article
- Integration of active human beta-galactosidase gene (100 kb) into genome using HSV/AAV amplicon vector.Gene therapy · 2007Article
- Mutant torsinA interferes with protein processing through the secretory pathway in DYT1 dystonia cells.Proceedings of the National Academy of Sciences of the United States of America · 2007Article
- Efficient site-specific integration of large transgenes by an enhanced herpes simplex virus/adeno-associated virus hybrid amplicon vector.Journal of virology · 2006Article
- Stable secondary structure near the nicking site for adeno-associated virus type 2 Rep proteins on human chromosome 19.Journal of virology · 2005Article
- Characterization of the mouse adeno-associated virus AAVS1 ortholog.Journal of virology · 2004Article
- HSV-1 amplicon vector-mediated expression of ATM cDNA and correction of the ataxia-telangiectasia cellular phenotype.Gene therapy · 2003Article
Corrections and comments
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Authors and funding
6 authors at 5 institutions in 1 country.
Funding
Abstract
Herpes simplex virus type 1/adeno-associated virus (HSV/AAV) rep(+) hybrid amplicon vectors containing AAV inverted terminal repeats (ITRs) and rep gene sequences can mediate site-specific integration into the human genome. In this study, we have generated and characterized the first transgenic mice that bear the full-length (8.2 kb) human AAVS1 locus. Immortalized mouse embryonic fibroblasts from this mouse line were transduced with the rep(+), rep(-) (containing only ITRs flanking the transgene) hybrid amplicon vectors, and the standard amplicon vector to determine stable integration frequency and the site of integration. Transduction of transgenic fibroblasts resulted in a 10-fold higher stable integration frequency with rep(+) hybrid amplicon vector than with rep(-) or standard amplicon vectors. Southern blot analysis of genomic DNA from transgenic cells stably transduced with the rep(+) hybrid amplicon vector revealed site-specific integration of transgenes at the AAVS1 locus in 50% of clones. Some site-specific and random integration events were limited to the ITR-flanked transgene cassette. In contrast, transduction of transgenic mouse cells with the rep(-) or standard amplicon vectors resulted in random integrations of the entire rep(-) hybrid amplicon or amplicon DNA that were incorporated into the host genome as a concatenate of various sizes. These results demonstrate for the first time that the genome of transgenic mice bearing the human AAVS1 locus serves as a platform for site-specific integration of AAV ITR-flanked transgene cassettes within the hybrid amplicon vector in the presence of Rep.
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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.