Trial reportPloS one2013
Production of factor VIII by human liver sinusoidal endothelial cells transplanted in immunodeficient uPA mice.
Trial report in PloS one, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 38 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
38 citing papers in PubMed, 58 citations in OpenAlex.
- Investigative reporting on factor VIII whereabouts.Blood advances · 2026Article
- Endothelial Cell Therapy for the Acute and Chronic Liver Disease.Seminars in liver disease · 2026Review
- Self-organization of sinusoidal vessels in pluripotent stem cell-derived human liver bud organoids.Nature biomedical engineering · 2025Article
- FVIII Trafficking Dynamics Across Subcellular Organelles Using CRISPR/Cas9 Specific Gene Knockouts.International journal of molecular sciences · 2025Article
- Dissecting endothelial cell heterogeneity with new tools.Cell regeneration (London, England) · 2025Review
- Aspects of zone-like identity and holotomographic tracking of human stem cell-derived liver sinusoidal endothelial cells.Frontiers in cell and developmental biology · 2025Article
- Efficient generation of liver sinusoidal endothelial-like cells secreting coagulation factor VIII from human induced pluripotent stem cells.Molecular therapy. Methods & clinical development · 2024Article
- Mice Engrafted with Human Liver Cells.Seminars in liver disease · 2024Review
- Fetal liver CD34Stem cell research & therapy · 2024Article
- A Monocytic Barrier to the Humanization of Immunodeficient Mice.Current stem cell research & therapy · 2024Review
- Generation of functional liver sinusoidal endothelial-like cells from human bone marrow-derived mesenchymal stem cells.Regenerative therapy · 2023Article
- Therapeutic potential of fetal liver cell transplantation in hemophilia A mice.Haematologica · 2023Article
- CD203c is expressed by human fetal hepatoblasts and distinguishes subsets of hepatoblastoma.Frontiers in oncology · 2023Article
- Antibody screening data of human midgestation liver cells with a focus on hematopoietic, liver sinusoidal endothelial, and hepatoblast cell-populations.BMC research notes · 2022Article
- Case Report: A Child With Hemophilia A Serves as Donor for Hematopoietic Stem Cell Transplantation to Cure His Brother's Severe Aplastic Anemia.Pathology oncology research : POR · 2022Article
- Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device.Molecular therapy. Methods & clinical development · 2021Article
- Resolving the graft ischemia-reperfusion injury during liver transplantation at the single cell resolution.Cell death & disease · 2021Article
- A cell based assay for evaluating binding and uptake of an antibody using hepatic nonparenchymal cells.Scientific reports · 2021Article
- Restoration of FVIII Function and Phenotypic Rescue in Hemophilia A Mice by Transplantation of MSCs Derived FromFrontiers in cell and developmental biology · 2021Article
- Flow-Cytometry Platform for Intracellular Detection of FVIII in Blood Cells: A New Tool to Assess Gene Therapy Efficiency for Hemophilia A.Molecular therapy. Methods & clinical development · 2020Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 2 institutions in 1 country.
Funding
Abstract
Liver sinusoidal endothelial cells (LSECs) form a semi-permeable barrier between parenchymal hepatocytes and the blood. LSECs participate in liver metabolism, clearance of pathological agents, immunological responses, architectural maintenance of the liver and synthesis of growth factors and cytokines. LSECs also play an important role in coagulation through the synthesis of Factor VIII (FVIII). Herein, we phenotypically define human LSECs isolated from fetal liver using flow cytometry and immunofluorescence microscopy. Isolated LSECs were cultured and shown to express endothelial markers and markers specific for the LSEC lineage. LSECs were also shown to engraft the liver when human fetal liver cells were transplanted into immunodeficient mice with liver specific expression of the urokinase-type plasminogen activator (uPA) transgene (uPA-NOG mice). Engrafted cells expressed human Factor VIII at levels approaching those found in human plasma. We also demonstrate engraftment of adult LSECs, as well as hepatocytes, transplanted into uPA-NOG mice. We propose that overexpression of uPA provides beneficial conditions for LSEC engraftment due to elevated expression of the angiogenic cytokine, vascular endothelial growth factor. This work provides a detailed characterization of human midgestation LSECs, thereby providing the means for their purification and culture based on their expression of CD14 and CD32 as well as a lack of CD45 expression. The uPA-NOG mouse is shown to be a permissive host for human LSECs and adult hepatocytes, but not fetal hepatoblasts. Thus, these mice provide a useful model system to study these cell types in vivo. Demonstration of human FVIII production by transplanted LSECs encourages further pursuit of LSEC transplantation as a cellular therapy for the treatment of hemophilia A.
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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.