Correction of murine beta-thalassemia by gene transfer into the germ line

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Science  12 Sep 1986:
Vol. 233, Issue 4769, pp. 1192-1194
DOI: 10.1126/science.3461564


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.