The Progress in Treatment of β-Thalassemia and Research on Animal Models of the Disease

β-thalassemia is one of the most prevalent single-gene recessive disorders worldwide, characterized by the impaired synthesis of β-globin chains, which leads to ineffective erythropoiesis and results in anemia and iron overload, along with various complications. Therefore, establishing animal models that closely resemble β-thalassemia is essential for studying the pathogenesis and treatment of this disease. This article reviews the molecular mechanisms underlying β-thalassemia; highlights the research advancements of several common clinical treatment methods; and provides a summary of prevalent animal models of β-thalassemia, including mice, rabbits, and cynomolgus monkeys. Although the mouse model reproduces certain pathological characteristics of β-thalassemia, it is limited in its ability to correct specific gene mutations, making it less effective for certain aspects of the disease. In contrast, the rabbit model, which more closely resembles human physiology, offers an improved approximation. Furthermore, the cynomolgus monkey model outperforms both the mouse and rabbit models in exhibiting the severe phenotype of β-thalassemia, thanks to its notable genetic and physiological similarities to humans. This article aims to provide a comprehensive reference for researchers conducting animal experimental studies in relation to β-thalassemia.