Challenging the Erythropoiesis Paradigm in β-Thalassemia

β-thalassemia is a genetic disorder due to defective β-globin synthesis that results in severe anemia. A hallmark of the disease is the massive expansion of the erythroid compartment associated with enhanced but ineffective erythropoiesis. Whether this result in remodeling and homeostatic changes of the bone marrow (BM) niche and what are the consequences on disease pathophysiology is unclear and remains understudied.

Through HSCT experiments, the authors demonstrated how an improved BM microenvironment can positively affect hematopoietic stem cell (HSC) behavior and ameliorate their functions.