ArticleGene therapy2011
Long-term phenotypic correction in factor IX knockout mice by using ΦC31 integrase-mediated gene therapy.
Article in Gene therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 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.
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Who cites it
14 citing papers in PubMed, 36 citations in OpenAlex.
- Transformative approaches in hemophilia management: from traditional therapies to prenatal stem cell treatment.Frontiers in bioengineering and biotechnology · 2025Review
- Global mapping of binding sites for phic31 integrase in transgenic maden-darby bovine kidney cells using ChIP-seq.Hereditas · 2019Article
- Review
- Ultrasound-targeted hepatic delivery of factor IX in hemophiliac mice.Gene therapy · 2016Article
- Long-term and efficient expression of human β-globin gene in a hematopoietic cell line using a new site-specific integrating non-viral system.Gene therapy · 2015Article
- Liver-targeted gene therapy: Approaches and challenges.Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society · 2015Review
- Gene therapy for hemophilia.Frontiers in bioscience (Landmark edition) · 2015Review
- Intragenic integration in DLC1 sustains factor VIII expression in primary human cells without insertional oncogenicity.Gene therapy · 2014Article
- Hybrid lentivirus-phiC31-int-NLS vector allows site-specific recombination in murine and human cells but induces DNA damage.PloS one · 2014Article
- Recombinase-mediated reprogramming and dystrophin gene addition in mdx mouse induced pluripotent stem cells.PloS one · 2014Article
- Establishment of stable reporter expression for in vivo imaging of nuclear factor-κB activation in mouse liver.Theranostics · 2013Article
- Bacteria-human somatic cell lateral gene transfer is enriched in cancer samples.PLoS computational biology · 2013Article
- Animal models of hemophilia.Progress in molecular biology and translational science · 2012Review
- Site-specific recombinase strategy to create induced pluripotent stem cells efficiently with plasmid DNA.Stem cells (Dayton, Ohio) · 2011Article
Corrections and comments
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Authors and funding
6 authors at 2 institutions in 1 country.
Funding
Abstract
Hemophilia B, a hereditary bleeding disorder caused by a deficiency of coagulation factor IX (FIX), is an excellent candidate for gene therapy. However, to date, success in hemophilia gene therapy clinical trials has been limited due to failure to achieve or sustain therapeutic levels of factor expression. The ΦC31 integrase system efficiently integrates plasmid DNA carrying a transgene and an attB site into a limited number of endogenous pseudo attP sites in mammalian genomes, leading to robust, sustained transgene expression. A strategy utilizing plasmid DNA integrated with ΦC31 integrase may offer a facile and safe alternative for sustained human FIX (hFIX) expression. Hydrodynamic tail vein injection was used for delivery of plasmids encoding ΦC31 integrase and hFIX to the liver of FIX knockout mice. We demonstrated prolonged therapeutic levels of hFIX in this knockout mouse model of hemophilia B over a 6-month time course when ΦC31 integrase was used. Additionally, we observed sustained FIX activity in plasma and phenotypic correction of bleeding after tail clip in ΦC31-treated mice. In the livers that received integrase, we also demonstrated prolonged hFIX expression in hepatocytes by immunohistochemistry and documented sequence-specific genomic integration of the hFIX plasmid. These studies suggest the possibility that a similar approach in large animals and humans could lead to a simple and successful gene therapy for hemophilia.
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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.