Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression

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...

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Detalles Bibliográficos
Autores principales: 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, Miccio, Annarita
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9636226/
https://www.ncbi.nlm.nih.gov/pubmed/36333351
http://dx.doi.org/10.1038/s41467-022-34493-1

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