Preclinical transfusion-dependent humanized mouse model of β thalassemia major

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Preclinical transfusion-dependent humanized mouse model of thalassemia major

A preclinical humanized mouse model of 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 and globin genes were replaced with adult human globin genes ( 2 1) and a human fetal to adult hemoglobin (Hb)–switching cassette ( HPFH 0), respectively. Similar to human infants with CA, fully humaniz...

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renal hemosiderosis among iranian transfusion dependent β-thalassemia major patients

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ژورنال

عنوان ژورنال: Blood

سال: 2009

ISSN: 0006-4971,1528-0020

DOI: 10.1182/blood-2008-12-197012