Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells.
Pavani G, Fabiano A, Laurent M, Amor F, Cantelli E, Chalumeau A, Maule G, Tachtsidi A, Concordet JP, Cereseto A, Mavilio F, Ferrari G, Miccio A, Amendola M.
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Blood Adv. 2021 Mar 9;5(5):1137-1153. doi: 10.1182/bloodadvances.2020001996.
Blood Adv. 2021.
PMID: 33635334
Free PMC article.