Article

Identification of novel HPFH-like mutations by CRISPR base editing that elevates the expression of fetal hemoglobin

2020-07-01

Abstract excerpt

<h4>ABSTRACT</h4> Switching hemoglobin synthesis from defective adult beta-globin to fetal gamma-globin is an effective strategy for the treatment of beta-hemoglobinopathies. Fetal hemoglobin expression is down-regulated in the postnatal period due to the interplay of transcription regulators with the HBG promoters. However, in the hereditary persistence of fetal hemoglobin (HPFH) condition, naturally occurring...

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Literature Corpus work: 057bf200-e4a4-58bd-b800-fadd4203ce80
DOI: 10.1101/2020.06.30.178715

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