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Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

Leslie Weber Giacomo Frati Tristan Felix Giulia Hardouin Antonio Casini Clara Wollenschlaeger Vasco Meneghini Cecile Masson Anne de Cian Anne Chalumeau Fulvio Mavilio Mario Amendola Isabelle Andre-Schmutz Anna Cereseto Wassim El Nemer Jean-Paul Concordet Carine Giovannangeli 1 Marina Cavazzana Annarita Miccio 
1 STRING - Structure et Instabilité des Génomes
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https://hal-mnhn.archives-ouvertes.fr/mnhn-03099960
Contributor : Carine Giovannangeli Connect in order to contact the contributor
Submitted on : Wednesday, January 6, 2021 - 1:47:01 PM
Last modification on : Wednesday, May 11, 2022 - 10:00:05 AM

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MNHN | CNRS | INC-CNRS | SORBONNE-UNIVERSITE | SU-TI

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Leslie Weber, Giacomo Frati, Tristan Felix, Giulia Hardouin, Antonio Casini, et al.. Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype. Science Advances , American Association for the Advancement of Science (AAAS), 2020, 6 (7), pp.eaay9392. ⟨10.1126/sciadv.aay9392⟩. ⟨mnhn-03099960⟩

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