Large-scale discovery of potent, compact and erythroid specific enhancers for gene therapy vectors.

Nat Commun · 2025 · PMC12064758 · PMID 40346084

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marginally significantno p-value reported

Use of PVT1 enhancer displayed consistently robust HbF and superior induction in both populations over μLCR, while HbF increase with μLCR was either insignificant (nucleated) or marginally significant (enucleated) over untransduced (Fig. 6b ).

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