Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

Disorders caused by abnormal β- globin, such as β- thalassaemia, are the most prevalent inherited disorders worldwide. For treatment, many patients are dependent on blood transfusions; thus far the only cure has involved matched transplantation of haematopoietic stem cells. Here it is shown that lentiviral β- globingene transfer can be an effective substitute for regular transfusions in a patient with severe β- thalassaemia.