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Science. 1997 Oct 31;278(5339):876-8.

Transgenic knockout mice with exclusively human sickle hemoglobin and sickle cell disease.

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Human Genome Center and Department of Subcellular Structure, Lawrence Berkeley National Laboratory, 1 Cyclotron Road (MS 74-157), University of California, Berkeley, CA 94720, USA.


To create mice expressing exclusively human sickle hemoglobin (HbS), transgenic mice expressing human alpha-, gamma-, and betaS-globin were generated and bred with knockout mice that had deletions of the murine alpha- and beta-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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