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

A murine β-thalassemia was corrected by the transfer of cloned β-globin genes into the mouse germ line. The cloned mouse β^maj-globin gene or the cloned human β-globin gene was introduced into mice deficient in β-globin synthesis because of a deletion of the β_maj-globin gene. Both introduced genes produced functional β-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.