Reactivation of γ-globin expression using a minicircle DNA system to treat β-thalassemia.
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DOI: 10.1016/j.gene.2022.146289
Abstract: Reactivation of fetal hemoglobin by editing the B-cell lymphoma/leukemia 11A (BCL11A) erythroid enhancer is an effective gene therapy for β-thalassemia. Using the CRISPR/Cas9 system, fetal γ-globin expression can be robustly reactivated to mitigate the clinical… read more here.
Keywords: dna; globin expression; expression; reactivation globin ... See more keywords