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Psatha, Nikoletta; Reik, Andreas; Phelps, Susan; Zhou, Yuanyue; Dalas, Demetri; Yannaki, Evangelia; Levasseur, Dana N.; Urnov, Fyodor D.; Holmes, Michael C.; Papayannopoulou, Thalia
Molecular therapy. Methods & clinical development, 09/2018, Letnik: 10Journal Article
In the present report, we carried out clinical-scale editing in adult mobilized CD34+ hematopoietic stem and progenitor cells (HSPCs) using zinc-finger nuclease-mediated disruption of BCL11a to upregulate the expression of γ-globin (fetal hemoglobin). In these cells, disruption of the erythroid-specific enhancer of the BCL11A gene increased endogenous γ-globin expression to levels that reached or exceeded those observed following knockout of the BCL11A coding region without negatively affecting survival or in vivo long-term proliferation of edited HSPCs and other lineages. In addition, BCL11A enhancer modification in mobilized CD34+ cells from patients with β-thalassemia major resulted in a readily detectable γ-globin increase with a preferential increase in G-gamma, leading to an improved phenotype and, likely, a survival advantage for maturing erythroid cells after editing. Furthermore, we documented that both normal and β-thalassemia HSPCs not only can be efficiently expanded ex vivo after editing but can also be successfully edited post-expansion, resulting in enhanced early in vivo engraftment compared with unexpanded cells. Overall, this work highlights a novel and effective treatment strategy for correcting the β-thalassemia phenotype by genome editing.
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JCR | SNIP | JCR | SNIP | JCR | SNIP | JCR | SNIP |
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