COUP-TFII regulates hemoglobin switching by activating the BCL11A-XL repressor Lin28B and directly binding δ and β globin promoters in fetal versus adult erythroid cells

The reactivation of fetal globin genes is the most promising treatment for β-hemoglobinopathies. This implies the reversal of the naturally occurring hemoglobin switching. Here, we show that expression of the orphan nuclear receptor COUP-TFII in adult HUDEP2 erythroid precursor cells activates γ-glo...

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Bibliographic Details
Published in:Haematologica (Roma) Vol. 999; no. 1
Main Authors: Frigo, Carlotta, Pastori, Valentina, Zambanini, Gianluca, Fabiano, Martina, Ahmed, Sajeela, Citterio, Elisabetta, Cantù, Claudio, Ronchi, Antonella Ellena
Format: Journal Article
Language:English
Published: Italy Ferrata Storti Foundation 27.11.2025
ISSN:1592-8721, 0390-6078, 1592-8721
Online Access:Get full text
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Summary:The reactivation of fetal globin genes is the most promising treatment for β-hemoglobinopathies. This implies the reversal of the naturally occurring hemoglobin switching. Here, we show that expression of the orphan nuclear receptor COUP-TFII in adult HUDEP2 erythroid precursor cells activates γ-globin (HbF) at the expense of β-adult globin by specific occupation of the "adult" δ-β-region within the β-locus. Notably, although COUPT-FII and the main γ-globin repressor BCL11A-XL share a similar DNA binding consensus and a large number of chromatin targets, including the locus control region of the β-locus itself, they bind differentially to the γ and β promoters, eliciting an opposite transcriptional outcome. In addition, we find that COUP-TFII activates LinN28B, a known posttranscriptional repressor of BCL11A-XL. Our work identifies a molecular mechanism that could be leveraged to increase γ-globin levels in patients affected by β-hemoglobinopathies.
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ISSN:1592-8721
0390-6078
1592-8721
DOI:10.3324/haematol.2025.288485