Journal
COOLEY'S ANEMIA: NINTH SYMPOSIUM
Volume 1202, Issue -, Pages 45-51Publisher
WILEY-BLACKWELL
DOI: 10.1111/j.1749-6632.2010.05547.x
Keywords
thalassemia; HPFH; transfusion; mouse model; hemoglobin switching
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Funding
- National Institutes of Health [R01 HL072351, R01 HL073440, T32 GM008111]
- Carmichael Scholarships
- NATIONAL HEART, LUNG, AND BLOOD INSTITUTE [R01HL073440, R01HL072351] Funding Source: NIH RePORTER
- NATIONAL INSTITUTE OF GENERAL MEDICAL SCIENCES [T32GM008111] Funding Source: NIH RePORTER
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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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