Article

Base editing of β <sup>0</sup> thalassemia mutations as a therapeutic strategy for β-hemoglobinopathies: efficacy and genotoxicity studies

2025-08-23

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 editors (ABE) variants to precisely correct some of the most prevalent and severe β-thalassemia-causing mutations in the β-globin gene. Efficien...

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Literature Corpus work: a6d4444a-ff60-5475-903e-507b1f01a2ea
DOI: 10.1101/2025.08.21.671453

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