Platelet-targeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A

M. Du, Lily; Nurden, Paquita; T. Nurden, Alan; C. Nichols, Timothy; A. Bellinger, Dwight; S. Jensen, Eric; L. Haberichter, Sandra; P. Merricks, Elizabeth; A. Raymer, Robin; Fang, Juan; B. Koukouritaki, Sevasti; M. Jacobi, Paula; Hawkins, Troy; Cornetta, Kenneth; Shi, Qizhen; A. Wilcox, David

doi:10.1038/ncomms3773

Public

Platelet-targeted gene therapy with human factor VIII establishes haemostasis in dogs with haemophilia A

Published in Nature Communications • Nov 19, 2013

Authors:

Lily M. Du

Paquita Nurden

Alan T. Nurden

Abstract

It is essential to improve therapies for controlling excessive bleeding in patients with haemorrhagic disorders. As activated blood platelets mediate the primary response to vascular injury, we hypothesize that storage of coagulation Factor VIII within platelets may provide a locally inducible treat...

Subject

Haemophilia

Platelet

Genetic enhancement

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