Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia

β-Thalassemia is one of the most common inherited anemias, with no effective cure for most patients. The pathophysiology reflects an imbalance between α- and β-globin chains with an excess of free α-globin chains causing ineffective erythropoiesis and hemolysis. When α-thalassemia is co-inherited wi...

Ausführliche Beschreibung