Factoring in new gene therapy treatments for hemophilia A

Science Translational Medicine
10 January 2018   Vol 10, Issue 423
http://stm.sciencemag.org/

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Factoring in new gene therapy treatments for hemophilia A
By Leah C. Byrne
Science Translational Medicine10 Jan 2018 Full Access
Delivery of a modified factor VIII gene leads to dramatic improvement of severe hemophilia A in human trials.