… in the β-globin (HBB) gene and affect millions of people worldwide. Ex vivo gene correction
in patient-derived haematopoietic stem cells … Here we present a CRISPR/Cas9 gene-editing …

… (A to T) in the β-globin (HBB) gene leading to a Glu6Val amino acid … gene can be effectively
edited in human hematopoietic stem and progenitor cells (HSPCs) using the CRISPR/Cas9 …

… Overall, we described an innovative CRISPR/Cas9 approach to improve α/β globin
imbalance in thalassemic HSPCs, paving the way for novel therapeutic strategies for β-thal. …

… including the δ- and β-globin genes and the putative intergenic … β-globin expression in an
adult erythroid cell line 23 and in healthy donor and SCD hematopoietic stem/progenitor cell …

… Editing the β-globin locus in hematopoietic stem cells is an … for gene therapy of β-thalassemia
and sickle cell disease. Using the CRISPR/Cas9 system, we genetically modified human …

… CRISPR/Cas9-mediated beta-globin (HBB) gene correction … validated in human hematopoietic
stem and progenitor cells (… is that the frequencies of gene targeting in this manuscript are …

… Allogeneic transplantation of bone marrow-derived hematopoietic stem cell (HSCs) at … /42)
of the human β-globin gene represents the most common β-thalassemia mutations [8]. …

… gene activation that runs along the β-globin gene cluster during development in a 5′ to 3′
direction, leading to the expression of the ε-gene (… Aiming for a more efficient CRISPR/Cas9 …

… of CRISPR/Cas9 genome editing of primary human hematopoietic stem/progenitor (CD34+)
cells to … f α-and β-globin gene expression normalized to the expression of RPL13A and α/…

… first time the correction, by CRISPR-Cas9 gene editing, of the β … β-globin genes after
CRISPR-Cas9 correction of the β 0 39-thalassemia mutation performed on erythroid precursor cells …