ArticleStem cells (Dayton, Ohio)2009
Busulfan produces efficient human cell engraftment in NOD/LtSz-Scid IL2Rgamma(null) mice.
Article in Stem cells (Dayton, Ohio), 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 50 papers, 2 of them syntheses that pooled it.
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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
50 citing papers in PubMed, 2 syntheses or guidelines pooled it, 66 citations in OpenAlex.
- The Coming of Age of Preclinical Models of MDS.Frontiers in oncology · 2022Pooled it
- Development of humanized mouse with patient-derived xenografts for cancer immunotherapy studies: A comprehensive review.Cancer science · 2021Pooled it
- Beyond the blood-brain barrier: humanised mice, the missing link in glioblastoma research.Oncogene · 2026Review
- Comparative analysis of NSG and NBSGW mice for preclinical evaluation of gene-modified human hematopoietic stem and progenitor cells.Stem cell research & therapy · 2026Article
- Generation of NOD SCID mice with near-complete deletions of Il2rg and Prkdc for human cancer and HSC engraftment.Transgenic research · 2025Article
- Post-transplant G-CSF impedes engraftment of gene-edited human hematopoietic stem cells by exacerbating p53-mediated DNA damage response.Cell stem cell · 2025Article
- Article
- Animal model considerations for chordoma research: reproducing the tumor microenvironmentFrontiers in oncology · 2024Review
- An overview of mouse models of hepatocellular carcinoma.Infectious agents and cancer · 2023Review
- Review
- Modeling the Tumor Microenvironment and Cancer Immunotherapy in Next-Generation Humanized Mice.Cancers · 2023Review
- Neuronal nitric oxide synthase is required for erythropoietin stimulated erythropoiesis in mice.Frontiers in cell and developmental biology · 2023Article
- Improved engraftment and therapeutic efficacy by human genome-edited hematopoietic stem cells with Busulfan-based myeloablation.Molecular therapy. Methods & clinical development · 2022Article
- Article
- Perspectives on Non-BLT Humanized Mouse Models for Studying HIV Pathogenesis and Therapy.Viruses · 2021Review
- Establishment of Humanized Mice for the Study of HBV.Frontiers in immunology · 2021Review
- Building the Next Generation of Humanized Hemato-Lymphoid System Mice.Frontiers in immunology · 2021Review
- BCL11A enhancer-edited hematopoietic stem cells persist in rhesus monkeys without toxicity.The Journal of clinical investigation · 2020Article
- Preclinical platform for long-term evaluation of immuno-oncology drugs using hCD34+ humanized mouse model.Journal for immunotherapy of cancer · 2020Article
- Immunoresponse to Gene-Modified Hematopoietic Stem Cells.Molecular therapy. Methods & clinical development · 2020Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 1 institution in 1 country.
Funding
Abstract
Xenografting immunodeficient mice after low-dose irradiation has been used as a surrogate human hematopoietic stem cell (HSC) assay; however, irradiation requires strict and meticulous animal support and can produce significant mortality rates, limiting the usefulness of this model. In this work, we examined the use of parenteral busulfan as an alternative conditioning agent. Busulfan led to dose-dependent human HSC engraftment in NOD/LtSz-scid/IL2Rgamma(null) mice, with marked improvement in survival rates. Terminally differentiated B and T lymphocytes made up most of the human CD45+ cells observed during the initial 5 weeks post-transplant when unselected cord blood (CB) products were infused, suggesting derivation from existing mature elements rather than HSCs. Beyond 5 weeks, CD34+-enriched products produced and sustained superior engraftment rates compared with unselected grafts (CB CD34+, 65.8% +/- 5.35%, vs. whole CB, 4.27% +/- 0.67%, at 24 weeks). CB CD34+ group achieved significantly higher levels of engraftment than mobilized CD34+-enriched peripheral blood (PB CD34+). At 8 weeks, all leukocyte subsets were detected, yet human red blood cells (RBCs) were not observed. Transfused human red cells persisted in the chimeric mice for up to 3 days; an accompanying rise in total bilirubin suggested hemolysis as a contributing factor to their clearance. Recipient mouse-derived human HSCs had the capacity to form erythroid colonies in vitro at various time points post-transplant in the presence of human transferrin (Tf). When human Tf was administered singly or in combination with anti-CD122 antibody and human cytokines, up to 0.1% human RBCs were detectable in the peripheral blood. This long evasive model should prove valuable for the study of human erythroid 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.