title: Knockout-transgenic mouse model of sickle cell disease.
abstract: When transgenic mice that expressed human sickle hemoglobin were mated with mice having knockout mutations of the mouse alpha- and beta-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.
authors.affiliation: Department of Biochemistry and Molecular Genetics, Schools of Medicine and Dentistry, University of Alabama at Birmingham, Birmingham, AL 35294, USA.
authors.forename: T M
authors.initials: TM
authors.lastname: Ryan
authors.forename: D J
authors.initials: DJ
authors.lastname: Ciavatta
authors.forename: T M
authors.initials: TM
authors.lastname: Townes
mesh_terms: D000755:Anemia, Sickle Cell / Q000097:blood / Q000235:genetics / Q000473:pathology; D000818:Animals; D002851:Chromatography, High Pressure Liquid; D003433:Crosses, Genetic; D004195:Disease Models, Animal; D004912:Erythrocytes / Q000473:pathology; D005914:Globins / Q000235:genetics; D006451:Hemoglobin, Sickle / Q000235:genetics; D006454:Hemoglobins / Q000235:genetics; D006801:Humans; D051379:Mice; D008810:Mice, Inbred C57BL; D018345:Mice, Knockout; D008822:Mice, Transgenic
chemical_list: D006451:Hemoglobin, Sickle; D006454:Hemoglobins; D005914:Globins