Editors' ChoiceGene Therapy

Factoring in new gene therapy treatments for hemophilia A

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Science Translational Medicine  10 Jan 2018:
Vol. 10, Issue 423, eaar7511
DOI: 10.1126/scitranslmed.aar7511

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Abstract

Delivery of a modified factor VIII gene leads to dramatic improvement of severe hemophilia A in human trials.

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