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...
मुख्य लेखकों: | 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 |
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स्वरूप: | Journal article |
प्रकाशित: |
Springer Nature
2017
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समान संसाधन
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Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia
द्वारा: Sachith Mettananda, और अन्य
प्रकाशित: (2017-09-01) -
Understanding α-globin gene regulation and implications for the treatment of β-thalassemia
द्वारा: Mettananda, S, और अन्य
प्रकाशित: (2016) -
Selective silencing of α-globin by the histone demethylase inhibitor IOX1: A potentially new pathway for treatment of β-thalassemia
द्वारा: Mettananda, S, और अन्य
प्रकाशित: (2016) -
P104: BASE EDITING REPAIRS THE HBE MUTATION RESTORING THE PRODUCTION OF NORMAL GLOBIN CHAINS IN SEVERE HBE/β-THALASSEMIA PATIENT HEMATOPOIETIC STEM AND ERYTHROID CELLS
द्वारा: M Badat, और अन्य
प्रकाशित: (2022-01-01) -
P104: Base editing repairs the HbE mutation restoring the production of normal globin chains in severe HbE/β-thalassemia patient hematopoietic stem and erythroid cells
द्वारा: Badat, M, और अन्य
प्रकाशित: (2022)