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Disruption of the BCL11A Erythroid Enhancer Reactivates Fetal Hemoglobin in Erythroid Cells of Patients with β-Thalassemia Major

Nikoletta Psatha, Andreas Reik, Susan Phelps, Yuanyue Zhou, Demetri Dalas, Evangelia Yannaki, Dana N. Levasseur, Fyodor D. Urnov, Michael C. Holmes, Thalia Papayannopoulou

Molecular Therapy — Methods & Clinical Development · 2018 · 111 citations · 46 references

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Abstract

engraftment compared with unexpanded cells. Overall, this work highlights a novel and effective treatment strategy for correcting the β-thalassemia phenotype by genome editing.

References

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