Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression
Antoniou and colleagues used base editing to generate a variety of mutations inducing γ-globin and rescue the β-hemoglobinopathy phenotype. This strategy was safe and effective in long-term repopulating hematopoietic stem/progenitor cells.
Bibliographic Details
| Main Authors: |
Panagiotis Antoniou,
Giulia Hardouin,
Pierre Martinucci,
Giacomo Frati,
Tristan Felix,
Anne Chalumeau,
Letizia Fontana,
Jeanne Martin,
Cecile Masson,
Megane Brusson,
Giulia Maule,
Marion Rosello,
Carine Giovannangeli,
Vincent Abramowski,
Jean-Pierre de Villartay,
Jean-Paul Concordet,
Filippo Del Bene,
Wassim El Nemer,
Mario Amendola,
Marina Cavazzana,
Anna Cereseto,
Oriana Romano,
Annarita Miccio |
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| Format: | Article
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| Language: | English |
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| Published: |
Nature Portfolio
2022-11-01
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| Series: | Nature Communications
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| Online Access: | https://doi.org/10.1038/s41467-022-34493-1
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