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...

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Main Authors:	Mettananda, S, Fisher, C, Hay, D, Badat, M, Quek, L, Clark, K, Hublitz, P, Downes, D, Kerry, J, Gosden, M, Telenius, J, Sloane-Stanley, J, Faustino, P, Coelho, A, Doondeea, J, Usukhbayar, B, Sopp, P, Sharpe, J, Hughes, J, Vyas, P, Gibbons, R, Higgs, D
格式:	Journal article
出版:	Springer Nature 2017

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

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