Nature Communications (Nov 2022)
Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression
- 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
Affiliations
- Panagiotis Antoniou
- Université Paris Cité, Imagine Institute, Laboratory of chromatin and gene regulation during development, INSERM UMR 1163
- Giulia Hardouin
- Université Paris Cité, Imagine Institute, Laboratory of chromatin and gene regulation during development, INSERM UMR 1163
- Pierre Martinucci
- Université Paris Cité, Imagine Institute, Laboratory of chromatin and gene regulation during development, INSERM UMR 1163
- Giacomo Frati
- Université Paris Cité, Imagine Institute, Laboratory of chromatin and gene regulation during development, INSERM UMR 1163
- Tristan Felix
- Université Paris Cité, Imagine Institute, Laboratory of chromatin and gene regulation during development, INSERM UMR 1163
- Anne Chalumeau
- Université Paris Cité, Imagine Institute, Laboratory of chromatin and gene regulation during development, INSERM UMR 1163
- Letizia Fontana
- Université Paris Cité, Imagine Institute, Laboratory of chromatin and gene regulation during development, INSERM UMR 1163
- Jeanne Martin
- Université Paris Cité, Imagine Institute, Laboratory of chromatin and gene regulation during development, INSERM UMR 1163
- Cecile Masson
- Bioinformatics Platform, Imagine Institute
- Megane Brusson
- Université Paris Cité, Imagine Institute, Laboratory of chromatin and gene regulation during development, INSERM UMR 1163
- Giulia Maule
- CIBIO, University of Trento
- Marion Rosello
- Sorbonne Université, INSERM, CNRS, Institut de la Vision
- Carine Giovannangeli
- INSERM U1154, CNRS UMR7196, Museum National d’Histoire Naturelle
- Vincent Abramowski
- Université Paris Cité, Imagine Institute, Laboratory of genome dynamics in the immune system, INSERM UMR 1163
- Jean-Pierre de Villartay
- Université Paris Cité, Imagine Institute, Laboratory of genome dynamics in the immune system, INSERM UMR 1163
- Jean-Paul Concordet
- INSERM U1154, CNRS UMR7196, Museum National d’Histoire Naturelle
- Filippo Del Bene
- Sorbonne Université, INSERM, CNRS, Institut de la Vision
- Wassim El Nemer
- Établissement Français du Sang, UMR 7268
- Mario Amendola
- Genethon
- Marina Cavazzana
- Biotherapy Department and Clinical Investigation Center, Assistance Publique Hopitaux de Paris, INSERM
- Anna Cereseto
- CIBIO, University of Trento
- Oriana Romano
- Department of Life Sciences, University of Modena and Reggio Emilia
- Annarita Miccio
- Université Paris Cité, Imagine Institute, Laboratory of chromatin and gene regulation during development, INSERM UMR 1163
- DOI
- https://doi.org/10.1038/s41467-022-34493-1
- Journal volume & issue
-
Vol. 13,
no. 1
pp. 1 – 22
Abstract
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.
