Transgenic Knockout Mice with Exclusively Human Sickle Hemoglobin and Sickle Cell Disease

Abstract

To create mice expressing exclusively human sickle hemoglobin (HbS), transgenic mice expressing human α-, γ-, and β S -globin were generated and bred with knockout mice that had deletions of the murine α- and β-globin genes. These sickle cell mice have the major features (irreversibly sickled red cells, anemia, multiorgan pathology) found in humans with sickle cell disease and, as such, represent a useful in vivo system to accelerate the development of improved therapies for this common genetic disease.