ArticleProceedings of the National Academy of Sciences of the United States of America2006
Correction of hemophilia as a proof of concept for treatment of monogenic diseases by fetal spleen transplantation.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2006. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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Who cites it
16 citing papers in PubMed, 34 citations in OpenAlex.
- Factor VIII originates primarily from anatomically distinct subsets of liver sinusoidal endothelial cells.Blood advances · 2026Article
- Genomic Designs of rAAVs Contribute to Pathological Changes in the Livers and Spleens of Mice.Advances in cell and gene therapy · 2022Article
- Characterization and visualization of murine coagulation factor VIII-producing cells in vivo.Scientific reports · 2021Article
- Exploring the Complex Role of Coagulation Factor VIII in Chronic Liver Disease.Cellular and molecular gastroenterology and hepatology · 2021Review
- Human immunodeficiency virus infection induces lymphoid fibrosis in the BM-liver-thymus-spleen humanized mouse model.JCI insight · 2018Article
- Preconditioning allows engraftment of mouse and human embryonic lung cells, enabling lung repair in mice.Nature medicine · 2015Article
- Extrahepatic sources of factor VIII potentially contribute to the coagulation cascade correcting the bleeding phenotype of mice with hemophilia A.Haematologica · 2015Article
- Progress and challenges in the development of a cell-based therapy for hemophilia A.Journal of thrombosis and haemostasis : JTH · 2014Review
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- Advanced therapies for the treatment of hemophilia: future perspectives.Orphanet journal of rare diseases · 2012Review
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- Enhancement of pig embryonic implants in factor VIII KO mice: a novel role for the coagulation cascade in organ size control.PloS one · 2009Article
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- Embryonic pig pancreatic tissue for the treatment of diabetes in a nonhuman primate model.Proceedings of the National Academy of Sciences of the United States of America · 2009Article
- Transplanted endothelial cells repopulate the liver endothelium and correct the phenotype of hemophilia A mice.The Journal of clinical investigation · 2008Article
Corrections and comments
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Authors and funding
10 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Previous clinical attempts to correct genetic deficiencies such as hemophilia or Gaucher disease by transplantation of allogeneic spleen were associated with aggressive graft versus host disease, mediated by mature T cells derived from the donor spleen. We show that a fetal pig spleen harvested at the embryonic day 42 stage, before the appearance of T cells, exhibited optimal growth potential upon transplantation into SCID mice, and the growing tissue expressed factor VIII. Transplantation of embryonic day 42 spleen tissue into hemophilic SCID mice led to complete alleviation of hemophilia within 2-3 months after transplant, as demonstrated by tail bleeding and by assays for factor VIII blood levels. These results provide a proof of principle to the concept that transplantation of a fetal spleen, obtained from a developmental stage before the appearance of T cells, could provide a novel treatment modality for genetic deficiencies of an enzyme or a factor that can be replaced by the growing spleen tissue.
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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.