We found a match
Your institution may have rights to this item. Sign in to continue.
- Title
Humanized mouse models of Cooley's anemia: correct fetal-to-adult hemoglobin switching, disease onset, and disease pathology.
- Authors
Huo, Yongliang; McConnell, Sean C.; Liu, Shanrun; Zhang, Tingting; Yang, Rui; Ren, Jinxiang; Ryan, Thomas M.
- Abstract
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.
- Subjects
THALASSEMIA; HEMOGLOBINS; GLOBIN gene expression; CELLULAR therapy; THERAPEUTIC use of iron chelates
- Publication
Annals of the New York Academy of Sciences, 2010, Vol 1202, Issue 1, p45
- ISSN
0077-8923
- Publication type
Article
- DOI
10.1111/j.1749-6632.2010.05547.x