Phenotypic correction of von Willebrand disease type 3 blood-derived endothelial cells with lentiviral vectors expressing von Willebrand factor

Von Willebrand disease (VWD) is an inherited bleeding disorder, caused by quantitative (type 1 and 3) or qualitative (type 2) defects in von Willebrand factor (VWF). Gene therapy is an appealing strategy for treatment of VWD because it is caused by a single gene defect and because VWF is secreted in...

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Détails bibliographiques
Auteurs principaux: De Meyer, Simon F., Vanhoorelbeke, Karen, Chuah, Marinee K., Pareyn, Inge, Gillijns, Veerle, Hebbel, Robert P., Collen, Désiré, Deckmyn, Hans, VandenDriessche, Thierry
Format: Artigo
Langue:Inglês
Publié: The American Society of Hematology 2006
Sujets:
Hemostasis, Thrombosis, and Vascular Biology
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC1895808/
https://ncbi.nlm.nih.gov/pubmed/16478886
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2005-09-3605
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