Molecular BiologyNature MedicinePeer-reviewed
Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells
M. Kyle Cromer, Joab Camarena, Renata M. Martin…·Matthew Hebden Porteus
This study replaces the HBA1 gene with HBB in β-thalassemia-derived HSPCs using CRISPR, normalizing globin ratios and restoring functional hemoglobin.
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