Knockout-Transgenic Mouse Model of Sickle Cell Disease

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Science  31 Oct 1997:
Vol. 278, Issue 5339, pp. 873-876
DOI: 10.1126/science.278.5339.873

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When transgenic mice that expressed human sickle hemoglobin were mated with mice having knockout mutations of the mouse α- and β-globin genes, animals were produced that synthesized only human hemoglobin in adult red blood cells. Similar to many human patients with sickle cell disease, the mice developed a severe hemolytic anemia and extensive organ pathology. Numerous sickled erythrocytes were observed in peripheral blood. Although chronically anemic, most animals survived for 2 to 9 months and were fertile. Drug and genetic therapies can now be tested in this mouse model of sickle cell disease.

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