ArticleCell reports2018
Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX.
Article in Cell reports, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
26 citing papers in PubMed, 38 citations in OpenAlex.
- Article
- Article
- Stem cell therapy: a novel frontier in the treatment of liver fibrosis/cirrhosis.Stem cell research & therapy · 2025Review
- Application of Induced Pluripotent Stem Cells (iPSCs) in Hereditary and Viral Diseases of the Liver: Modeling and Treatment.International journal of molecular sciences · 2025Review
- Article
- Development of iPSC-derived FIX-secreting hepatocyte sheet as a novel treatment tool for hemophilia B treatment.Stem cell research & therapy · 2025Article
- Can hemophilia be cured? It depends on the definition.Research and practice in thrombosis and haemostasis · 2024Review
- Innovations in RNA therapy for hemophilia.Blood · 2023Review
- Gene Therapy Approaches for the Treatment of Hemophilia B.International journal of molecular sciences · 2023Review
- CRISPR-Mediated In Situ Introduction or Integration ofInternational journal of molecular sciences · 2023Article
- "iPSC-derived liver organoids and inherited bleeding disorders: Potential and future perspectives".Frontiers in physiology · 2023Review
- Article
- Review
- 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
- Review
- Review
- In vivo enrichment of genetically manipulated platelets for murine hemophilia B gene therapy.Journal of cellular physiology · 2021Article
- Differentiation of human induced pluripotent stem cells into erythroid cells.Stem cell research & therapy · 2020Review
- CRISPR/Cas9-mediated gene correction in hemophilia B patient-derived iPSCs.International journal of hematology · 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
8 authors at 4 institutions in 1 country.
Funding
Abstract
Hemophilia B is an ideal target for gene- and cell-based therapies because of its monogenic nature and broad therapeutic index. Here, we demonstrate the use of cell therapy as a potential long-term cure for hemophilia B in our FIX-deficient mouse model. We show that transplanted, cryopreserved, cadaveric human hepatocytes remain functional for more than a year and secrete FIX at therapeutic levels. Hepatocytes from different sources (companies and donors) perform comparably in curing the bleeding defect. We also generated induced pluripotent stem cells (iPSCs) from two hemophilia B patients and corrected the disease-causing mutations in them by two different approaches (mutation specific and universal). These corrected iPSCs were differentiated into hepatocyte-like cells (HLCs) and transplanted into hemophilic mice. We demonstrate these iPSC-HLCs to be viable and functional in mouse models for 9-12 months. This study aims to establish the use of cells from autologous and heterologous sources to treat hemophilia B.
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What OpenQuestion holds
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.