Novel Therapeutic Advances in β-Thalassemia
“Sapientia maior est quam fatum” (Wisdom is greater than fate)
DOI:
https://doi.org/10.69898/jhtm.35.2025.275741Keywords:
β-thalassemia, Novel therapy, Ineffective erythropoiesis, Hematopoietic stem cell transplantation, Gene therapyAbstract
β-thalassemia is among the most prevalent genetic disorders worldwide, characterized by impaired β-globin production, resulting in ineffective erythropoiesis, the hallmark of the disease. Beyond the conventional treatments, including regular blood transfusions and iron chelation therapy, recent advances in understanding the pathophysiology of β-thalassemia have led to the development of novel therapeutic strategies. Currently, several therapeutic strategies addressing the underlying mechanisms of disease, which include both curative approaches, such as hematopoietic stem cell transplantation (HSCT) and gene therapy, and pharmacological approaches modifying the molecular basis of disease, have been established. While these novel therapies have demonstrated clinically significant efficacy in various studies, there are several challenges in response variability among the patients, accessibility, and long-term safety outcomes which have been mentioned and remained in areas of improvement. In this review, we provide an overview of the novel therapeutic advances in β-thalassemia focusing on the underlying disease mechanisms, their potential therapeutic strategies, and current available treatments that will expand the therapeutic options for thalassemic patients.
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