Base editing of β0-thalassemia mutations as a therapeutic strategy for severe β-hemoglobinopathies.

doi:10.1126/scitranslmed.adt8617

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Article

Base editing of β0-thalassemia mutations as a therapeutic strategy for severe β-hemoglobinopathies.

Science translational medicine - 26 Nov 2025

Hardouin Giulia, Martinucci Pierre, Scaramuzza Samantha, Antoniou Panagiotis, Corradi Federico, Tachtsidi Alexandra, Corre Guillaume, Mombled Margaux, Chermont Jessika, Manceau Sandra, Rouillon Cécile, Masson Cécile, Joseph Laure, Thuret Isabelle, Badens Catherine, Szepetowsky Sarah, Allemand Eric, Amendola Mario, Romano Oriana, Ferrari Giuliana, Miccio Annarita

Abstract excerpt

Gene therapy has emerged as a promising curative treatment for β-hemoglobinopathies, the most common genetic disorders worldwide. However, current approved approaches still have some limitations in terms of safety and efficacy. Here, we used highly processive adenine base editor (ABE) variants to precisely correct some of the most prevalent and severe β-thalassemia-causing mutations in the β-globin-encoding HBB...

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PMID: 41296828
DOI: 10.1126/scitranslmed.adt8617

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