ArticleProceedings of the National Academy of Sciences of the United States of America2009
Phenotypic correction of murine hemophilia A using an iPS cell-based therapy.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 104 papers.
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Who cites it
104 citing papers in PubMed, 257 citations in OpenAlex.
- Therapeutic advances in hemophilia: from molecular innovation to patient-centered global care.Frontiers in medicine · 2025Review
- Recent Advances in Gene Therapy for Hemophilia: Projecting the Perspectives.Biomolecules · 2024Review
- 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
- Global Trends of Stem Cell Precision Medicine Research (2018-2022): A Bibliometric Analysis.Frontiers in surgery · 2022Review
- Single-cell sequencing reveals the potential oncogenic expression atlas of human iPSC-derived cardiomyocytes.Biology open · 2021Article
- Endothelial cells derived from patients' induced pluripotent stem cells for sustained factor VIII delivery and the treatment of hemophilia A.Stem cells translational medicine · 2020Article
- Bioengineering hemophilia A-specific microvascular grafts for delivery of full-length factor VIII into the bloodstream.Blood advances · 2019Article
- ssODN-Mediated In-Frame Deletion with CRISPR/Cas9 Restores FVIII Function in Hemophilia A-Patient-Derived iPSCs and ECs.Molecular therapy. Nucleic acids · 2019Article
- Patient-Specific iPSC-Derived Endothelial Cells Provide Long-Term Phenotypic Correction of Hemophilia A.Stem cell reports · 2018Article
- Evaluation of ex vivo produced endothelial progenitor cells for autologous transplantation in primates.Stem cell research & therapy · 2018Article
- In Utero Transplantation of Placenta-Derived Mesenchymal Stromal Cells for Potential Fetal Treatment of Hemophilia A.Cell transplantation · 2018Article
- Induced pluripotent stem cell: A headway in reprogramming with promising approach in regenerative biology.Veterinary world · 2017Review
- An effective ex-vivo approach for inducing endothelial progenitor cells from umbilical cord blood CD34Stem cell research & therapy · 2017Article
- Regenerative Medicine and the Biliary Tree.Seminars in liver disease · 2017Review
- Hepatocyte Transplantation: Cell Sheet Technology for Liver Cell Transplantation.Current transplantation reports · 2017Review
- Induced Pluripotent Stem Cell for the Study and Treatment of Sickle Cell Anemia.Stem cells international · 2017Article
- TGF-β1 along with other platelet contents augments Treg cells to suppress anti-FVIII immune responses in hemophilia A mice.Blood advances · 2016Article
- In situ genetic correction of F8 intron 22 inversion in hemophilia A patient-specific iPSCs.Scientific reports · 2016Article
- Hedgehog Signaling Overcomes an EZH2-Dependent Epigenetic Barrier to Promote Cholangiocyte Expansion.PloS one · 2016Article
- iPSCs: A Minireview from Bench to Bed, including Organoids and the CRISPR System.Stem cells international · 2016Review
44 more citing papers are in PubMed but not listed here.
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Authors and funding
7 authors at 1 institution in 1 country.
Funding
Abstract
Hemophilia A is caused by mutations within the Factor VIII (FVIII) gene that lead to depleted protein production and inefficient blood clotting. Several attempts at gene therapy have failed for various reasons-including immune rejection. The recent generation of induced pluripotent stem (iPS) cells from somatic cells by the ectopic expression of 3 transcription factors, Oct4, Sox2, and Klf4, provides a means of circumventing the immune rejection barrier. To date, iPS cells appear to be indistinguishable from ES cells and thus provide tremendous therapeutic potential. Here we prepared murine iPS cells from tail-tip fibroblasts and differentiated them to both endothelial cells and endothelial progenitor cells by using the embryoid body differentiation method. These iPS cells express major ES cell markers such as Oct4, Nanog, SSEA-1, alkaline phosphatase, and SALL4. Endothelial/endothelial progenitor cells derived from iPS cells expressed cell-specific markers such as CD31, CD34, and Flk1 and secreted FVIII protein. These iPS-derived cells were injected directly into the liver of irradiated hemophilia A mice. At various times after transplantation (7-90 days) hemophilia A mice and their control mice counterparts were challenged by a tail-clip bleeding assay. Nontransplanted hemophilia A mice died within a few hours, whereas transplanted mice survived for more than 3 months. Plasma FVIII levels increased in transplanted hemophilia A mice during this period to 8% to 12% of wild type and corrected the hemophilia A phenotype. Our studies provide additional evidence that iPS cell therapy may be able to treat human monogenetic disorders in the future.
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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.