Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin.

Shows that the use of recombinant lentiviruses enables efficient transfer and faithful integration of the human beta-globin gene together with large segments of its locus control region. Speculation that the stable introduction of a functional beta-globin gene in hemotopoietic stem cells could be a powerful approach to treat beta-thalassemia and sickle-cell disease; Findings of experiments with beta-thalassemic mice; Conclusion that such therapy should be of benefit to patients with severe defects in hemoglobin production.