Article

Identification of small molecule agonists of fetal hemoglobin expression for the treatment of sickle cell disease

2024-07-03

Abstract excerpt

Induction of fetal hemoglobin (HbF) has been shown to be a viable therapeutic approach to treating sickle cell disease and potentially other β-hemoglobinopathies. To identify targets and target-modulating small molecules that enhance HbF expression, we engineered a human umbilical-derived erythroid progenitor reporter cell line (HUDEP2_HBG1_HiBiT) by genetically tagging a HiBiT peptide to the carboxyl (C)-terminus...

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Literature Corpus work: 56cd512a-fbbb-5373-8452-fe378f4cabdf
DOI: 10.1101/2024.07.01.601536

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