High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation
iScience(2022)
Abstract
•The gene editing protocol corrects the sickle mutation in ∼30% of engrafting cells•Random assortment of engrafting stem cell clones without clonal dominance was shown•Corrected erythroid cells are preferentially enriched compared with unedited cells
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Key words
genetics,molecular genetics,genetic engineering
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