Recent trends and innovative strategies in the curative approach for β-thalassemia: a current prospect and future perspective.

β-Thalassemia, a major genetic disease affecting the global population, has seen new strategies being pursued in terms of curative attempts directed at the specific gene defect present. Iron chelation therapy and lifelong blood transfusions have historically been used to treat β-thalassemia, a severe hereditary hemoglobin condition marked by decreased or missing β-globin chain synthesis. These methods have significantly increased patient survival, but they do not offer a permanent solution and are linked to serious side effects and a high cost of care. With a focus on hematopoietic stem cell transplantation (HSCT), lentiviral gene-addition treatment, and future genome-editing techniques, this study outlines new and creative methods to long-term disease correction in β-thalassemia. Although allogeneic HSCT is still a proven treatment option, its use is constrained by the availability of donors, graft-related issues, and transplant-related hazards. Autologous hematopoietic stem cell-based gene therapy has become a significant substitute, provided the possibility of long-term transfusion independence while prevented graft-versus-host disease. With the creation of exagamglogene autotemcel (exa-cel; Casgevy), which reactivates fetal hemoglobin expression through modification of the BCL11A erythroid enhancer and has shown significant rates of sustained transfusion independence in eligible patients with transfusion-dependent β-thalassemia, CRISPR/Cas9-based genome editing has recently marked a significant clinical milestone. Notwithstanding these encouraging developments, myeloablative conditioning, long-term safety and durability, high treatment costs, complicated manufacturing requirements, and restricted accessibility continue to be major obstacles, especially in low- and middle-income nations. All things considered, the therapeutic landscape for β-thalassemia is changing from lifelong supportive management to potentially curative and transformative interventions; however, the wider application of these cutting-edge therapies depends on ongoing long-term evaluation and efforts to increase affordability and global accessibility.