Correction of murine beta-thalassemia by gene transfer into the germ line.
A murine beta-thalassemia was corrected by the transfer of cloned beta-globin genes into the mouse germ line. The cloned mouse beta maj-globin gene or the cloned human beta-globin gene was introduced into mice deficient in beta-globin synthesis because of a deletion of the beta maj-globin gene. Both introduced genes produced functional beta-globin chains, leading to a reduction in one case, and elimination in another case, of the anemia and associated abnormalities of the red blood cells.
Costantini
F
F
Chada
K
K
Magram
J
J
D000818:Animals; D005260:Female; D005796:Genes; D005818:Genetic Engineering; D005854:Germ Cells / Q000502:physiology*; D005914:Globins / Q000235:genetics; D006454:Hemoglobins / Q000235:genetics; D006720:Homozygote; D006801:Humans; D008297:Male; D051379:Mice; D013789:Thalassemia / Q000235:genetics / Q000628:therapy*
D006454:Hemoglobins; D005914:Globins