A study on gene therapy in beta-thalassemia was recently published in the New England Journal of Medicine, a genetic disease characterized by severe anemia that until now could only be treated by transfusion or bone marrow transplantation. In this study, the patients’ haematopoietic stem cells were taken and infected with a harmless virus called lentivirus, on which the gene encoding normal beta-globin was loaded. In this way the cells infected with this viral vector have been reinfused in the patients, thus restoring the normal functionality of the hemoglobin chains.
N Engl J Med. 2018 Apr 19;378(16):1479-1493. doi: 10.1056/NEJMoa1705342.
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia.
Thompson AA1, Walters MC1, Kwiatkowski J1, Rasko JEJ1, Ribeil JA1, Hongeng S1, Magrin E1, Schiller GJ1, Payen E1, Semeraro M1, Moshous D1, Lefrere F1, Puy H1, Bourget P1, Magnani A1, Caccavelli L1, Diana JS1, Suarez F1, Monpoux F1, Brousse V1, Poirot C1, Brouzes C1, Meritet JF1, Pondarré C1, Beuzard Y1, Chrétien S1, Lefebvre T1, Teachey DT1, Anurathapan U1, Ho PJ1, von Kalle C1, Kletzel M1, Vichinsky E1, Soni S1, Veres G1, Negre O1, Ross RW1, Davidson D1, Petrusich A1, Sandler L1, Asmal M1, Hermine O1, De Montalembert M1, Hacein-Bey-Abina S1, Blanche S1, Leboulch P1, Cavazzana M1.