Humanized mouse models of Cooley's anemia: correct fetal-to-adult hemoglobin switching, disease onset, and disease pathology
β 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...
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Published in | Annals of the New York Academy of Sciences Vol. 1202; no. 1; pp. 45 - 51 |
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Main Authors | , , , , , , |
Format | Journal Article |
Language | English |
Published |
Malden, USA
Blackwell Publishing Inc
01.08.2010
Wiley Subscription Services, Inc |
Subjects | |
Online Access | Get full text |
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Summary: | β 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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Bibliography: | ark:/67375/WNG-4FT0K8C0-T istex:AA444269C33AA5316819C1885FD87920F77D1EF9 ArticleID:NYAS5547 ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23 ObjectType-Article-2 ObjectType-Feature-1 |
ISSN: | 0077-8923 1749-6632 |
DOI: | 10.1111/j.1749-6632.2010.05547.x |