Abstract:
β-thalassemia is an inherited hemolytic anemia caused by mutations or deletions in the β-globin gene. Although conventional treatments such as blood transfusion, iron chelation therapy, and splenectomy can be lifesustaining, they are associated with limitations including poor quality of life, iron overload complications, and a substantial economic burden. In recent years, with the advent of breakthrough technologies and innovative drugs —such as gene editing, lentiviral vector-mediated gene addition, optimized haploidentical hematopoietic stem cell transplantation, and erythroid maturation agents—the field has entered a new era of precision and personalized medicine. This article systematically reviews the latest groundbreaking advances in the global treatment of β-thalassemia, with a focus on analyzing key clinical trial data and mechanisms of action of cutting-edge technologies like gene therapy. Furthermore, it discusses the optimization of personalized treatment strategies and future challenges within the context of clinical practice.