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This is a preprint. It has not yet been peer reviewed by a journal. The National Library of Medicine is running a pilot to include preprints that result from research funded by NIH in PMC and PubMed. Display options Abstract Gene editing the BCL11A erythroid enhancer is a validated approach to fetal hemoglobin (HbF) induction for β-hemoglobinopathy therapy, though heterogeneity in edit allele distribution and HbF response may impact its safety and efficacy.
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