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• W2751081544 abstract "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 with β-thalassemia, excess free α-globin chains are reduced significantly ameliorating the clinical severity. Here we demonstrate the use of CRISPR/Cas9 genome editing of primary human hematopoietic stem/progenitor (CD34+) cells to emulate a natural mutation, which deletes the MCS-R2 α-globin enhancer and causes α-thalassemia. When edited CD34+ cells are differentiated into erythroid cells, we observe the expected reduction in α-globin expression and a correction of the pathologic globin chain imbalance in cells from patients with β-thalassemia. Xenograft assays show that a proportion of the edited CD34+ cells are long-term repopulating hematopoietic stem cells, demonstrating the potential of this approach for translation into a therapy for β-thalassemia.β-thalassemia is characterised by the presence of an excess of α-globin chains, which contribute to erythrocyte pathology. Here the authors use CRISP/Cas9 to reduce α-globin expression in hematopoietic precursors, and show effectiveness in xenograft assays in mice." @default.
• W2751081544 created "2017-09-15" @default.
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• W2751081544 date "2017-09-04" @default.
• W2751081544 modified "2023-09-30" @default.
• W2751081544 title "Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia" @default.
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• W2751081544 doi "https://doi.org/10.1038/s41467-017-00479-7" @default.
• W2751081544 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/5583283" @default.
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