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Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells

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Giulia Pavani, Anna Fabiano, Marine Laurent, Fatima Amor, Erika Cantelli, Anne Chalumeau, Giulia Maule, Alexandra Tachtsidi, Jean-Paul Concordet, Anna Cereseto, Fulvio Mavilio, Giuliana Ferrari, Annarita Miccio, Mario Amendola
Journal: Blood Advances
Blood Adv (2021) 5 (5): 1137–1153.
https://doi.org/10.1182/bloodadvances.2020001996
Published: 2021
Includes: Supplemental data