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 w...
| المؤلفون الرئيسيون: | , , , , , , , , , , , , , , , , , , , , , |
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| التنسيق: | Journal article |
| منشور في: |
Springer Nature
2017
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