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In vivo HSC prime editing rescues sickle cell disease in a mouse model.
20
2023
-globin gene, CD46, Cas9, HSC, HSCs, S alleles, SCD, Sickle cell disease, Townes, adult, fetal globin, hematopoietic stem cells, hemoglobin, long-term repopulating HSCs, mice, mouse, prime editor, sickle, sickle cell disease, sickle hemoglobin, target site, the
Author NameAffiliation
Gregory A NewbyMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard
Gregory A NewbyHarvard University
Gregory A NewbyHoward Hughes Medical Institute, Harvard University
David R LiuMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard
David R LiuHarvard University
David R LiuHoward Hughes Medical Institute, Harvard University
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