Article
Identification of novel HPFH-like mutations by CRISPR base editing that elevate the expression of fetal hemoglobin
11 Feb 2022
Abstract excerpt
Naturally occurring point mutations in the HBG promoter switch hemoglobin synthesis from defective adult beta-globin to fetal gamma-globin in sickle cell patients with hereditary persistence of fetal hemoglobin (HPFH) and ameliorate the clinical severity. Inspired by this natural phenomenon, we tiled the highly homologous HBG proximal promoters using adenine and cytosine base editors that avoid the generation of...
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