A knockout of a transgenic mouse—animal models of sickle cell anemia

RL Nagel - New England Journal of Medicine, 1998 - Mass Medical Soc
RL Nagel
New England Journal of Medicine, 1998Mass Medical Soc
Animal models of sickle cell anemia can be used to improve understanding of the
pathophysiology of the disease, aid in the development of antisickling drugs, and test the
feasibility of gene therapy. The basis of these interesting models is transgenic technology, in
which foreign genes are introduced into the germ line of a mouse. With this method it is
possible to create mice that carry the mutant sickle, or β S, gene and a normal human α-
globin gene. In such animals, the human β S and α-globin genes code for sickle hemoglobin …
Animal models of sickle cell anemia can be used to improve understanding of the pathophysiology of the disease, aid in the development of antisickling drugs, and test the feasibility of gene therapy. The basis of these interesting models is transgenic technology, in which foreign genes are introduced into the germ line of a mouse. With this method it is possible to create mice that carry the mutant sickle, or βS, gene and a normal human α-globin gene. In such animals, the human βS and α-globin genes code for sickle hemoglobin (α2βS 2), but . . .
The New England Journal Of Medicine