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Storage and regulated secretion of factor VIII in blood outgrowth endothelial cells

By Maartje van den Biggelaar, Eveline A.M. Bouwens, Neeltje A. Kootstra, Robert P. Hebbel, Jan Voorberg and Koen Mertens


In this study, the authors explored the feasibility of blood outgrowth endothelial cells as a cellular FVIII delivery device with particular reference to long-term production levels, intracellular storage in Weibel-Palade bodies and agonist-induced regulated secretion. The findings of this ex vivo study open a new avenue towards gene therapy for hemophilia A

Topics: Original Article
Publisher: Ferrata Storti Foundation
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Provided by: PubMed Central
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