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Paper Details

Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells.
Nat Med
45
2021
-, -Thalassemia, -globin, -globin messenger RNA, Cas9, Edited HSPCs, HBA1, HBA1 gene, HBA1/2, HBB, HBB transgene, HSPCs, globin, globin messenger RNA, hematopoietic stem and progenitor cells, hemoglobin, mice, patient, patient-, patient-derived red blood cells, thalassemia, thalassemia-derived hematopoietic stem and progenitor cells
Author NameAffiliation
Ravindra MajetiStanford University
Ravindra MajetiInstitute of Stem Cell Biology and Regenerative Medicine, Stanford University
Vivien A SheehanBaylor College of Medicine
Matthew H PorteusStanford University
Matthew H PorteusInstitute of Stem Cell Biology and Regenerative Medicine, Stanford University
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