Gene therapy & β-thalassemia
It seems that the hope of gene therapy for transfusion-dependent β-thalassemia (TDT) is becoming so close. During the 62nd American Society of Hematology (ASH) annual conference, bluebird bio Inc presented a longe term data for their innovative and breakthrough gene therapy betibeglogene autotemcel gene therapy ( LentiGlobin™). Long-term follow-up showed that all patients who achieved transfusion independency remained …
