Key Points • PUM1, an RNA-binding protein, is a novel target of EKLF that binds to fetal γ-globin mRNA and impairs its stability and translation.• Elevated HbF levels are observed… Click to show full abstract
Key Points • PUM1, an RNA-binding protein, is a novel target of EKLF that binds to fetal γ-globin mRNA and impairs its stability and translation.• Elevated HbF levels are observed upon PUM1 knockdown ex vivo and in an individual harboring a novel PUM1 mutation in the RNA-binding domain.
               
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