ReviewJournal of thrombosis and haemostasis : JTH2014
Progress and challenges in the development of a cell-based therapy for hemophilia A.
Review in Journal of thrombosis and haemostasis : JTH, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 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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Who cites it
14 citing papers in PubMed, 31 citations in OpenAlex.
- Ectopic Expression of FVIII in HPCs and MSCs Derived from hiPSCs with Site-Specific Integration ofInternational journal of molecular sciences · 2022Article
- Comparison of different gene addition strategies to modify placental derived-mesenchymal stromal cells to produce FVIII.Frontiers in immunology · 2022Article
- Insights of CRISPR-Cas systems in stem cells: progress in regenerative medicine.Molecular biology reports · 2022Review
- High Mutational Heterogeneity, and New Mutations in the Human Coagulation Factor V Gene. Future Perspectives for Factor V Deficiency Using Recombinant and Advanced Therapies.International journal of molecular sciences · 2021Article
- Utilization of a novel Sendai virus vector in ex vivo gene therapy for hemophilia A.International journal of hematology · 2021Article
- Bone fragility in patients affected by congenital diseases non skeletal in origin.Orphanet journal of rare diseases · 2021Review
- Molecular Analysis of Fetal and Adult Primary Human Liver Sinusoidal Endothelial Cells: A Comparison to Other Endothelial Cells.International journal of molecular sciences · 2020Article
- Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII.Scientific reports · 2019Article
- Leucocyte and Platelet-rich Fibrin: a carrier of autologous multipotent cells for regenerative medicine.Journal of cellular and molecular medicine · 2018Article
- Novel therapies and current clinical progress in hemophilia A.Therapeutic advances in hematology · 2018Review
- Evaluation of high-fat high-fructose diet treatment in factor VIII (coagulation factor)-deficient mouse model.International journal of experimental pathology · 2018Article
- Human fetal liver cultures support multiple cell lineages that can engraft immunodeficient mice.Open biology · 2017Article
- Evaluation of the biological differences of canine and human factor VIII in gene delivery: implications in human hemophilia treatment.Gene therapy · 2016Article
- The search for the origin of factor VIII synthesis and its impact on therapeutic strategies for hemophilia A.Haematologica · 2015Article
Corrections and comments
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Authors and funding
3 authors at 2 institutions in 1 country.
Funding
Abstract
Hemophilia A results from an insufficiency of factor VIII (FVIII). Although replacement therapy with plasma-derived or recombinant FVIII is a life-saving therapy for hemophilia A patients, such therapy is a life-long treatment rather than a cure for the disease. In this review, we discuss the possibilities, progress, and challenges that remain in the development of a cell-based cure for hemophilia A. The success of cell therapy depends on the type and availability of donor cells, the age of the host and method of transplantation, and the levels of engraftment and production of FVIII by the graft. Early therapy, possibly even prenatal transplantation, may yield the highest levels of engraftment by avoiding immunological rejection of the graft. Potential cell sources of FVIII include a specialized subset of endothelial cells known as liver sinusoidal endothelial cells (LSECs) present in the adult and fetal liver, or patient-specific endothelial cells derived from induced pluripotent stem cells that have undergone gene editing to produce FVIII. Achieving sufficient engraftment of transplanted LSECs is one of the obstacles to successful cell therapy for hemophilia A. We discuss recent results from transplants performed in animals that show production of functional and clinically relevant levels of FVIII obtained from donor LSECs. Hence, the possibility of treating hemophilia A can be envisioned through persistent production of FVIII from transplanted donor cells derived from a number of potential cell sources or through creation of donor endothelial cells from patient-specific induced pluripotent stem cells.
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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.