Transgenic knockout mice with exclusively human sickle hemoglobin and sickle cell disease.
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.
Pászty
C
C
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_paszty@csa2.lbl.gov
Brion
C M
CM
Manci
E
E
Witkowska
H E
HE
Stevens
M E
ME
Mohandas
N
N
Rubin
E M
EM
D000755:Anemia, Sickle Cell / Q000235:genetics* / Q000473:pathology; D000818:Animals; D004195:Disease Models, Animal; D005260:Female; D005914:Globins / Q000235:genetics; D006451:Hemoglobin, Sickle / Q000235:genetics; D006801:Humans; D008297:Male; D051379:Mice; D008810:Mice, Inbred C57BL; D018345:Mice, Knockout; D008822:Mice, Transgenic
D006451:Hemoglobin, Sickle; D005914:Globins