Article

Genomic discovery and functional validation of MRP1 as a novel fetal hemoglobin modulator and potential therapeutic target in sickle cell disease

2023-03-15

Abstract excerpt

<h4>ABSTRACT</h4> Sickle cell disease (SCD) remains a major health burden with limited treatment options. Despite promising gene-editing clinical trials, there is an unmet need for cost-effective therapies. As induction of fetal hemoglobin (HbF) is an established therapeutic strategy for SCD, we conducted a genome-wide association study of circulating HbF levels in ~11,000 participants to identify further HbF modu...

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Literature Corpus work: 6b2a99fb-c94e-521b-86fe-13b8b8e56d6c
DOI: 10.1101/2023.03.14.23287244

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