NDLI: Preclinical transfusion-dependent humanized mouse model of β thalassemia major

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Preclinical transfusion-dependent humanized mouse model of β thalassemia major

Content Provider	Scilit
Author	Huo, Yongliang McConnell, Sean C. Ryan, Thomas M.
Copyright Year	2009
Description	A preclinical humanized mouse model of β thalassemia major or Cooley anemia (CA) was generated by targeted gene replacement of the mouse adult globin genes in embryonic stem cells. The mouse adult α and β globin genes were replaced with adult human α globin genes (α2α1) and a human fetal to adult hemoglobin (Hb)–switching cassette (γHPFHδβ0), respectively. Similar to human infants with CA, fully humanized mice survived postnatally by synthesizing predominantly human fetal Hb, HbF (α2γ2), with a small amount of human minor adult Hb, HbA2 (α2δ2). Completion of the human fetal to adult Hb switch after birth resulted in severe anemia marked by erythroid hyperplasia, ineffective erythropoiesis, hemolysis, and death. Similar to human patients, CA mice were rescued from lethal anemia by regular blood transfusion. Transfusion corrected the anemia and effectively suppressed the ineffective erythropoiesis, but led to iron overload. This preclinical humanized animal model of CA will be useful for the development of new transfusion and iron chelation regimens, the study of iron homeostasis in disease, and testing of cellular and genetic therapies for the correction of thalassemia.
Related Links	http://www.bloodjournal.org/content/bloodjournal/113/19/4763.full.pdf
Ending Page	4770
Page Count	8
Starting Page	4763
DOI	10.1182/blood-2008-12-197012
Journal	Blood
Issue Number	19
Volume Number	113
Language	English
Publisher	American Society of Hematology
Publisher Date	2009-05-07
Access Restriction	Open
Subject Keyword	Research and Experimental Medicine Anemia Iron Thalassemia Embryonic Stem Cells Mouse Model Human Genetic Therapies Hemolysis Blood Transfusion Globin Journal: Blood (Vol- 130, Issue- 19)
Content Type	Text
Resource Type	Article

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