Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivati...
Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression
About this item
Full title
Author / Creator
Antoniou, Panagiotis , Hardouin, Giulia , Martinucci, Pierre , Frati, Giacomo , Felix, Tristan , Chalumeau, Anne , Fontana, Letizia , Martin, Jeanne , Masson, Cecile , Brusson, Megane , Maule, Giulia , Rosello, Marion , Giovannangeli, Carine , Abramowski, Vincent , de Villartay, Jean-Pierre , Concordet, Jean-Paul , Del Bene, Filippo , El Nemer, Wassim , Amendola, Mario , Cavazzana, Marina , Cereseto, Anna , Romano, Oriana and Miccio, Annarita
Publisher
London: Nature Publishing Group UK
Journal title
Language
English
Formats
Publication information
Publisher
London: Nature Publishing Group UK
Subjects
More information
Scope and Contents
Contents
Sickle cell disease and β-thalassemia affect the production of the adult β-hemoglobin chain. The clinical severity is lessened by mutations that cause fetal γ-globin expression in adult life (i.e., the hereditary persistence of fetal hemoglobin). Mutations clustering ~200 nucleotides upstream of the
HBG
transcriptional start sites either redu...
Alternative Titles
Full title
Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression
Authors, Artists and Contributors
Author / Creator
Hardouin, Giulia
Martinucci, Pierre
Frati, Giacomo
Felix, Tristan
Chalumeau, Anne
Fontana, Letizia
Martin, Jeanne
Masson, Cecile
Brusson, Megane
Maule, Giulia
Rosello, Marion
Giovannangeli, Carine
Abramowski, Vincent
de Villartay, Jean-Pierre
Concordet, Jean-Paul
Del Bene, Filippo
El Nemer, Wassim
Amendola, Mario
Cavazzana, Marina
Cereseto, Anna
Romano, Oriana
Miccio, Annarita
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_313aa0fc4a794f6798f4ea808d498494
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_313aa0fc4a794f6798f4ea808d498494
Other Identifiers
ISSN
2041-1723
E-ISSN
2041-1723
DOI
10.1038/s41467-022-34493-1