ArticleAnnals of the New York Academy of Sciences2010
Humanized mouse models of Cooley's anemia: correct fetal-to-adult hemoglobin switching, disease onset, and disease pathology.
Article in Annals of the New York Academy of Sciences, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.
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Who cites it
10 citing papers in PubMed, 12 citations in OpenAlex.
- Understanding erythroid physiology and pathology in humanized mice: A closer look.British journal of haematology · 2025Review
- Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies.Pharmaceutics · 2022Review
- Minihepcidins improve ineffective erythropoiesis and splenomegaly in a new mouse model of adult β-thalassemia major.Haematologica · 2020Article
- In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques.Scientific reports · 2019Article
- Humanising the mouse genome piece by piece.Nature communications · 2019Review
- Article
- Allogeneic bone marrow transplant in the absence of cytoreductive conditioning rescues mice with β-thalassemia major.Blood advances · 2017Article
- Non-invasive MRI biomarkers for the early assessment of iron overload in a humanized mouse model of β-thalassemia.Scientific reports · 2017Article
- Recent trends in the gene therapy of β-thalassemia.Journal of blood medicine · 2015Review
- Human globin knock-in mice complete fetal-to-adult hemoglobin switching in postnatal development.Molecular and cellular biology · 2011Article
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Authors and funding
7 authors at 1 institution in 1 country.
Funding
Abstract
beta thalassemia major or Cooley's Anemia (CA) has been difficult to model in mice due to their lack of a fetal hemoglobin gene equivalent. This summary describes novel preclinical humanized mouse models of CA that survive on human fetal hemoglobin at birth and are blood-transfusion dependent for life upon completion of their human fetal-to-adult hemoglobin switch after birth. These CA models are the first to recapitulate the temporal onset of the disease in human patients. These novel humanized CA disease models are useful for the study of the regulation of globin gene expression, synthesis, and switching; examining the onset of disease pathology; development of transfusion and iron chelation therapies; induction of fetal hemoglobin synthesis; and the testing of novel genetic and cell-based therapies for the correction of thalassemia.
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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.