ArticleGene therapy2009
A 16-bp RBE element mediated Rep-dependent site-specific integration in AAVS1 transgenic mice for expression of hFIX.
Article in Gene therapy, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 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.
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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
7 citing papers in PubMed, 17 citations in OpenAlex.
- Residual DNA impurities in AAV vectors-nature and transcription.Molecular therapy. Methods & clinical development · 2025Article
- Recent Advances in CRISPR/Cas9 Delivery Approaches for Therapeutic Gene Editing of Stem Cells.Stem cell reviews and reports · 2023Review
- Viral Delivery Systems for CRISPR.Viruses · 2019Review
- Review
- Gene therapy for hemophilia B mice with scAAV8-LP1-hFIX.Frontiers of medicine · 2016Article
- Reply to "Wild-type AAV Insertions in Hepatocellular Carcinoma Do Not Inform Debate Over Genotoxicity Risk of Vectorized AAV".Molecular therapy : the journal of the American Society of Gene Therapy · 2016Article
- Structural Insights into the Assembly of the Adeno-associated Virus Type 2 Rep68 Protein on the Integration Site AAVS1.The Journal of biological chemistry · 2015Article
Corrections and comments
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Authors and funding
11 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
We earlier reported that, a 16 bp Rep-binding element (RBE) was sufficient for mediating Rep-dependent integration into AAVS1 in vitro. We explored here the potential use of this RBE in site-specific genome integration at the AAVS1 site in vivo using transgenic mice carrying the human AAVS1 locus in their genome. In the presence of a Rep-donor plasmid, an human blood coagulation factor IX (hFIX) expression plasmid (pRBE-CMV-hFIX) containing the 16 bp RBE was delivered to AAVS1 transgenic mice by hydrodynamic injection. Insertion of the transgene into the AAVS1 site of the mouse genome was confirmed by nested PCR at the junction of the plasmid/AAVS1 locus. Sequencing analysis found the site-specific insertion in four of seven animals injected with pRBE-CMV-hFIX but in none of the mice injected with pN2-CMV-hFIX, the control construct without the 16 bp RBE or with pRBE-CMV-hFIX plasmid but without co-expressing Rep. Plasma hFIX levels in pRBE-CMV-hFIX-injected animals were higher and lasted longer than in the pN2-CMV-hFIX control group. The levels of hFIX in pRBE-CMV-hFIX-injected animals were also significantly higher than in the control animals after partial hepatectomy (PH). These results showed that the 16 bp RBE could mediate the delivery of a therapeutic gene into the AAVS1 locus in a Rep-dependent, site-specific manner in vivo, suggesting its potential application in gene therapy.
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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.