ArticleThe Journal of clinical investigation2008
Transplanted endothelial cells repopulate the liver endothelium and correct the phenotype of hemophilia A mice.
Article in The Journal of clinical investigation, 2008. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 87 papers, 1 of them a synthesis that pooled it.
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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
87 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Methods for Isolation and Purification of Murine Liver Sinusoidal Endothelial Cells: A Systematic Review.PloS one · 2016Pooled it
- Production of factor VIII by human liver sinusoidal endothelial cells transplanted in immunodeficient uPA mice.PloS one · 2013Trial
- Factor VIII originates primarily from anatomically distinct subsets of liver sinusoidal endothelial cells.Blood advances · 2026Article
- Endothelial Cell Therapy for the Acute and Chronic Liver Disease.Seminars in liver disease · 2026Review
- Transformative approaches in hemophilia management: from traditional therapies to prenatal stem cell treatment.Frontiers in bioengineering and biotechnology · 2025Review
- Research progress in stem cell therapy for Wilson disease.Regenerative therapy · 2024Review
- Factor VIII promotes angiogenesis and vessel stability regulating extracellular matrix proteins.Haematologica · 2024Article
- Hemostasis and endothelial functionality: the double face of coagulation factors.Haematologica · 2024Review
- GP64-pseudotyped lentiviral vectors target liver endothelial cells and correct hemophilia A mice.EMBO molecular medicine · 2024Article
- Generation of functional liver sinusoidal endothelial-like cells from human bone marrow-derived mesenchymal stem cells.Regenerative therapy · 2023Article
- Improved intravenous lentiviral gene therapy based on endothelial-specific promoter-driven factor VIII expression for hemophilia A.Molecular medicine (Cambridge, Mass.) · 2023Article
- Therapeutic potential of fetal liver cell transplantation in hemophilia A mice.Haematologica · 2023Article
- Adeno-Associated Virus Gene Therapy for Hemophilia.Annual review of medicine · 2023Review
- Hepatocyte Transplantation Rebalances Cytokines for Hepatic Regeneration in Rats with Ataxia Telangiectasia Mutated Pathway-Related Acute Liver Failure.The American journal of pathology · 2023Article
- The Vascular Endothelium and Coagulation: Homeostasis, Disease, and Treatment, with a Focus on the Von Willebrand Factor and Factors VIII and V.International journal of molecular sciences · 2022Review
- In Vitro Conditioning of Adipose-Derived Mesenchymal Stem Cells by the Endothelial Microenvironment: Modeling Cell Responsiveness towards Non-Genetic Correction of Haemophilia A.International journal of molecular sciences · 2022Article
- Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates.Nature communications · 2022Article
- Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device.Molecular therapy. Methods & clinical development · 2021Article
- Hemophilia gene therapy: ushering in a new treatment paradigm?Hematology. American Society of Hematology. Education Program · 2021Review
- Deciphering the Ets-1/2-mediated transcriptional regulation of F8 gene identifies a minimal F8 promoter for hemophilia A gene therapy.Haematologica · 2021Article
27 more citing papers are in PubMed but not listed here.
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.
Funding
Abstract
Transplantation of healthy cells to repair organ damage or replace deficient functions constitutes a major goal of cell therapy. However, the mechanisms by which transplanted cells engraft, proliferate, and function remain unknown. To investigate whether host liver sinusoidal endothelium could be replaced with transplanted liver sinusoidal endothelial cells, we developed an animal model of tissue replacement that utilized a genetic system to identify transplanted cells and induced host-cell perturbations to confer a proliferative advantage to transplanted cells. Under these experimental conditions, transplanted cells engrafted efficiently and proliferated to replace substantial portions of the liver endothelium. Tissue studies demonstrated that transplanted cells became integral to the liver structure and reacquired characteristic endothelial morphology. Characterization of transplanted endothelial cells by membrane markers and studies of cellular function, including synthesis and release of coagulation factor VIII, demonstrated that transplanted cells were functionally intact. Further analysis showed that repopulation of the livers of mice that model hemophilia A with healthy endothelial cells restored plasma factor VIII activity and corrected their bleeding phenotype. Our studies therefore suggest that transplantation of healthy endothelial cells should be considered for cell therapy of relevant disorders and that endothelial reconstitution with transplanted cells may offer an excellent paradigm for defining organ-specific pathophysiological mechanisms.
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Registered trials
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