Stem cell treatment & Haemophilia

Written based on 20 research studies on PubMed, searched on 2026. nov. 11..

The claim

Posts online say that stem cell treatment helps with haemophilia.

Where research stands

We found 62 published papers on stem cell treatment and haemophilia. They were published between 2004 and 2025. None of them is a study in people. 18 were studies in animals, and 6 were only in cells in the lab. 16 papers are reviews that sum up other studies. 3 new studies in people are running or will start soon, say the trial registers.

• 0In people
• 18In animals
• 6Only in the lab

What the studies found

We have not summed up these studies in words yet. The most important papers are listed under Sources.

Verdict

Unproven means: it was only tested in animals or cells, or there is too little or mixed data from people. How we rate claims

Sources

1. Transformative approaches in hemophilia management: from traditional therapies to prenatal stem cell treatment.2025 · Front Bioeng Biotechnol · Review
2. Curative Therapies for Hemophilias and Hemoglobinopathies in Adults: Immune, Gene, and Stem Cell Approaches in a Global Context.2025 · Biomedicines · Review
3. Cartilage degeneration in hemophilic arthropathy: to focus on prevention or regeneration?2025 · Expert Rev Hematol · Review
4. What is the rationale for mesenchymal stromal cells based therapies in the management of hemophilic arthropathies?2024 · Osteoarthritis Cartilage · Review
5. [Post-transplantation lymphoproliferative disorder accompanies acquired hemophilia after haploid hematopoietic stem cell transplantation in a pediatric AML patient: a case report and literature review].2019 · Zhonghua Xue Ye Xue Za Zhi · Review
6. Induced Pluripotent Stem Cell Technology: A Paradigm Shift in Medical Science for Drug Screening and Disease Modeling.2017 · Curr Med Chem · Review
7. The bleeding edge: broadening horizons for hematopoietic stem cell therapies.2025 · Trends Mol Med · In animals
8. Human umbilical cord mesenchymal stem cell-based gene therapy for hemophilia B using scAAV-DJ/8-LP1-hFIXco transduction.2024 · Stem Cell Res Ther · In animals
9. PAM-flexible Cas9-mediated base editing of a hemophilia B mutation in induced pluripotent stem cells.2023 · Commun Med (Lond) · In animals
10. In Vitro FVIII-Encoding Transgenic Mesenchymal Stem Cells Maintain Successful Coagulation in FVIII-Deficient Plasma Mimicking Hemophilia A.2023 · Turk J Haematol · In animals
11. Therapeutic potential of fetal liver cell transplantation in hemophilia A mice.2023 · Haematologica · In animals
12. Non-genotoxic conditioning facilitates hematopoietic stem cell gene therapy for hemophilia A using bioengineered factor VIII.2021 · Mol Ther Methods Clin Dev · In animals
13. CRISPR/Cas9 Edition of the F9 Gene in Human Mesenchymal Stem Cells for Hemophilia B Therapy.2024 · Life (Basel) · In the lab
14. Generation an induced pluripotent stem cell line SXMUi001-A derived from a hemophilia B patient carries variant F9 c.223C＞T(p.R75X).2022 · Stem Cell Res · In the lab
15. Generation of induced pluripotent stem cell, BCHSCTi001-A, derived from a Hemophilia A patient with F8 (p. R391C) mutation.2021 · Stem Cell Res · In the lab
16. Generation of induced pluripotent stem cell GZLSL-i001-A derived from urine-derived cells of Hemophilia A patient with Inv22 mutation.2020 · Stem Cell Res · In the lab
17. Successful Hematopoietic Stem Cell Transplantation in a Child with Thalassemia major and Moderate Haemophilia A.2024 · Indian J Hematol Blood Transfus · Other paper
18. Delivery of Cas9-guided ABE8e into stem cells using poly(l-lysine) polypeptides for correction of the hemophilia-associated FIX missense mutation.2022 · Biochem Biophys Res Commun · Other paper
19. In Vitro Conditioning of Adipose-Derived Mesenchymal Stem Cells by the Endothelial Microenvironment: Modeling Cell Responsiveness towards Non-Genetic Correction of Haemophilia A.2022 · Int J Mol Sci · Other paper
20. Case Report: A Child With Hemophilia A Serves as Donor for Hematopoietic Stem Cell Transplantation to Cure His Brother's Severe Aplastic Anemia.2022 · Pathol Oncol Res · Other paper