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Science 31 October 1997:
Vol. 278. no. 5339, pp. 876 - 878
DOI: 10.1126/science.278.5339.876
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Reports

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

Chris Pászty, * Catherine M. Brion, Elizabeth Manci, H. Ewa Witkowska, Mary E. Stevens, Narla Mohandas, Edward M. Rubin

To create mice expressing exclusively human sickle hemoglobin (HbS), transgenic mice expressing human alpha -, gamma -, and beta S-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.

C. Pászty and N. Mohandas, 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.
C. M. Brion, M. E. Stevens, E. M. Rubin, Human Genome Center, Lawrence Berkeley National Laboratory, University of California, Berkeley, CA 94720, USA.
E. Manci, Centralized Pathology Unit for Sickle Cell Disease, University of South Alabama Doctors Hospital, Mobile, AL 36604, USA.
H. E. Witkowska, Children's Hospital Oakland Research Institute, Oakland, CA 94609, USA.
*   To whom correspondence should be addressed. E-mail: c_paszty{at}csa2.lbl.gov

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