Article

Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathies

2022-07-13

Abstract excerpt

Reactivation of fetal hemoglobin (HbF) is the commonly adapted strategy to ameliorate β-hemoglobinopathies. However, the continued production of defective adult hemoglobin (HbA) limits the HbF tetramer production affecting the therapeutic benefits. Here, we tested various deletional hereditary persistence of fetal hemoglobin (HPFH) mutations and identified a 11 kb sequence, encompassing Putative Repressor Region (...

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Identifiers and source

Literature Corpus work: eb286fb7-4ae1-51aa-b428-5e41f2dfd670
DOI: 10.1101/2022.07.11.499646

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