Editors' ChoiceGene Therapy

Gene transfer delivers (β-globin)

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Science Translational Medicine  02 May 2018:
Vol. 10, Issue 439, eaat8522
DOI: 10.1126/scitranslmed.aat8522

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Abstract

Transplantation of genetically engineered hematopoietic stem cells eliminates or reduces transfusion dependence in β-thalassemia.

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