Better to transfer than transfuse?

Abstract

Gene Therapy β-Thalassemia is a blood disease caused by mutations in the β-globin gene. β-Globin is a subunit of hemoglobin, an oxygen-carrying protein in red blood cells. Patients with β-thalassemia are severely anemic and require lifelong transfusions of red blood cells. In two clinical trials involving a total of 22 patients, Thompson et al. tested a gene therapy–based treatment that might eliminate the need for repeated transfusions. They isolated hematopoietic stem cells from the patients, used a lentiviral vector to transfer a “normal” copy of the β-globin gene into the cells, and then infused the modified cells back into the patients. After 26 months, the patients showed sustained expression of the transgenic hemoglobin, and nearly 70% of them no longer required transfusions. New Engl. J. Med. 378 , 1479 (2018).