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

Sickle cell disease (SCD) is caused by a single amino acid change in the adult hemoglobin (Hb) β chain that causes Hb polymerization and red blood cell (RBC) sickling. The co-inheritance of mutations causing fetal γ-globin production in adult life hereditary persistence of fetal Hb (HPFH) reduces th...

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Veröffentlicht in:Sci Adv
Hauptverfasser: Weber, Leslie, Frati, Giacomo, Felix, Tristan, Hardouin, Giulia, Casini, Antonio, Wollenschlaeger, Clara, Meneghini, Vasco, Masson, Cecile, De Cian, Anne, Chalumeau, Anne, Mavilio, Fulvio, Amendola, Mario, Andre-Schmutz, Isabelle, Cereseto, Anna, El Nemer, Wassim, Concordet, Jean-Paul, Giovannangeli, Carine, Cavazzana, Marina, Miccio, Annarita
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Association for the Advancement of Science 2020
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Research Articles
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7015694/
https://ncbi.nlm.nih.gov/pubmed/32917636
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/sciadv.aay9392
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