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Article

Targeting factor VIII expression to platelets for hemophilia A gene therapy does not induce an apparent thrombotic risk in mice.

Journal of thrombosis and haemostasis : JTH - 1 Jan 2017

Baumgartner C K, Mattson J G, Weiler H, Shi Q, Montgomery R R

Abstract excerpt

Essentials Platelet-Factor (F) VIII gene therapy is a promising treatment in hemophilia A. This study aims to evaluate if platelet-FVIII expression would increase the risk for thrombosis. Targeting FVIII expression to platelets does not induce or elevate thrombosis risk. Platelets expressing FVIII are neither hyper-activated nor hyper-responsive. SUMMARY: Background Targeting factor (F) VIII expression to...

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Identifiers

PMID: 27496751
DOI: 10.1111/jth.13436
PMCID: PMC5280575

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