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

Authors:
P√°szty C, Brion CM, Manci E, Witkowska HE, Stevens ME, Mohandas N, Rubin EM
Affiliation:
Journal:
Science (New York, N.Y.)

Abstract

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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