Editors' ChoiceSICKLE CELL DISEASE

Rounding up sickle cells with gene therapy

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Science Translational Medicine  15 Mar 2017:
Vol. 9, Issue 381, eaam9864
DOI: 10.1126/scitranslmed.aam9864

Abstract

A report of a patient treated with ex vivo lentiviral gene transfer to hematopoietic stem cells shows the promise of gene therapy for sickle cell anemia.

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