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

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Science  31 Oct 1997:
Vol. 278, Issue 5339, pp. 876-878
DOI: 10.1126/science.278.5339.876

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To create mice expressing exclusively human sickle hemoglobin (HbS), transgenic mice expressing human α-, γ-, and βS-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.

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