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; Marina Cavazzana; Annarita Miccio

doi:10.1126/sciadv.aay9392

Journal Articles Science Advances Year : 2020

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

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1

Leslie Weber

Function : Author

Giacomo Frati

Function : Author
PersonId : 771316
ORCID : 0000-0003-2703-3761

Tristan Felix

Function : Author

Giulia Hardouin

Function : Author

Antonio Casini

Function : Author

Clara Wollenschlaeger

Function : Author

Vasco Meneghini

Function : Author

Cecile Masson

Function : Author

Anne de Cian

Function : Author

Anne Chalumeau

Function : Author

Fulvio Mavilio

Function : Author

Mario Amendola

Function : Author
PersonId : 176487
IdHAL : mario-amendola
ORCID : 0000-0002-1188-8856
IdRef : 250171392

Isabelle Andre-Schmutz

Function : Author
PersonId : 745689
IdHAL : isabelleandreinsermfr
ORCID : 0000-0002-3905-9910
IdRef : 166097101

Anna Cereseto

Function : Author

Wassim El Nemer

Function : Author
PersonId : 752810
IdHAL : wassim-el-nemer
ORCID : 0000-0001-8184-427X
IdRef : 169097749

CV

Jean-Paul Concordet

Function : Author

Carine Giovannangeli

Function : Author
PersonId : 180503
IdHAL : carine-giovannangeli
ORCID : 0000-0002-7715-8864
IdRef : 111871220

Structure et Instabilité des Génomes

Marina Cavazzana

Function : Author
PersonId : 776350
ORCID : 0000-0002-0264-0891
IdRef : 084138653

Annarita Miccio

Function : Author

Domains

Life Sciences [q-bio] Biochemistry, Molecular Biology Life Sciences [q-bio]

Carine Giovannangeli : Connect in order to contact the contributor

https://hal-mnhn.archives-ouvertes.fr/mnhn-03099960

Submitted on : Wednesday, January 6, 2021-1:47:01 PM

Last modification on : Wednesday, May 11, 2022-10:00:05 AM

Dates and versions

mnhn-03099960 , version 1 (06-01-2021)

Identifiers

HAL Id : mnhn-03099960 , version 1
DOI : 10.1126/sciadv.aay9392
PUBMEDCENTRAL : PMC7015694

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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 , 2020, 6 (7), pp.eaay9392. ⟨10.1126/sciadv.aay9392⟩. ⟨mnhn-03099960⟩

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

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