Recent advances in the treatment of β-thalassemia: from traditional therapies to gene therapy

β-thalassemia is a monogenic hereditary disorder, with current treatments including blood transfusions, iron chelation therapy, and hematopoietic stem cell transplantation. Despite their benefits, these approaches have significant limitations. Recent advancements in pharmacological agents and gene therapy show promising therapeutic potential. This review systematically summarizes these emerging treatment modalities, offering clinical practitioners and researchers up-to-date insights. Initially, a comprehensive literature search was performed on PubMed using the keywords "thalassemia," "epidemiology," "treatment," "gene therapy," and "molecular mechanism". From approximately 180 articles published over the past decade, 89 relevant studies were included. This review covers both foundational knowledge of β-thalassemia and treatment advancements from the past five years. The findings provide a detailed overview of the molecular mechanisms and traditional treatments of β-thalassemia, with particular emphasis on recent pharmacotherapy and gene therapy advancements. Future research directions are also proposed. Novel pharmacological agents and gene therapy offer innovative avenues for managing β-thalassemia, presenting renewed hope for patients.