Molecular BiologybioRxivPreprint
Dual α-globin and truncated erythropoietin receptor knock-in restores hemoglobin production in α-thalassemia major-derived hematopoietic stem and progenitor cells
Simon N. Chu, Eric Soupène, Beeke Wienert…·M. Kyle Cromer
This paper describes a genome editing strategy that restores hemoglobin production in α-thalassemia major-derived hematopoietic stem and progenitor cells by integrating a functional α-globin gene and a truncated erythropoietin receptor.
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