[PDF] Gene Therapy for Von Willebrand Disease by Marinee Chuah, Inge Petrus, Thierry VandenDriessche · 10.1002/9781444329926.ch20 · OA.mg (2024)

Marinee Chuah,Inge Petrus,Thierry VandenDriessche

Chapter 20 Gene Therapy for Von Willebrand Disease Marinee K. L. Chuah PhD, Marinee K. L. Chuah PhD Flanders Institute for Biotechnology (VIB), Vesalius Research Center, University of Leuven, Leuven, Belgium Faculty of Medicine and Pharmacy, University Hospital Campus Jette, Free University of Brussels (VUB), Brussels, BelgiumSearch for more papers by this authorInge Petrus PhD, Inge Petrus PhD Flanders Institute for Biotechnology (VIB), Vesalius Research Center, University of Leuven, Leuven, BelgiumSearch for more papers by this authorThierry VandenDriessche PhD, Thierry VandenDriessche PhD Flanders Institute for Biotechnology (VIB), Vesalius Research Center, University of Leuven, Leuven, Belgium Faculty of Medicine and Pharmacy, University Hospital Campus Jette, Free University of Brussels (VUB), Brussels, BelgiumSearch for more papers by this author Marinee K. L. Chuah PhD, Marinee K. L. Chuah PhD Flanders Institute for Biotechnology (VIB), Vesalius Research Center, University of Leuven, Leuven, Belgium Faculty of Medicine and Pharmacy, University Hospital Campus Jette, Free University of Brussels (VUB), Brussels, BelgiumSearch for more papers by this authorInge Petrus PhD, Inge Petrus PhD Flanders Institute for Biotechnology (VIB), Vesalius Research Center, University of Leuven, Leuven, BelgiumSearch for more papers by this authorThierry VandenDriessche PhD, Thierry VandenDriessche PhD Flanders Institute for Biotechnology (VIB), Vesalius Research Center, University of Leuven, Leuven, Belgium Faculty of Medicine and Pharmacy, University Hospital Campus Jette, Free University of Brussels (VUB), Brussels, BelgiumSearch for more papers by this author Book Editor(s):Augusto B. Federici MD, Augusto B. Federici MD Division of Hematology and Transfusion Medicine, L. Sacco University Hospital, Department of Internal Medicine, University of Milan, Milan, ItalySearch for more papers by this authorChristine A. Lee MA, MD, DSc (Med), FRCP, FRCPath, FRCOGad eundem, Christine A. Lee MA, MD, DSc (Med), FRCP, FRCPath, FRCOGad eundem University of London, London, UKSearch for more papers by this authorErik E. Berntorp MD, PhD, Erik E. Berntorp MD, PhD Malmö Centre for Thrombosis and Haemostasis, Lund University, Skåne University Hospital, Malmö, SwedenSearch for more papers by this authorDavid Lillicrap MD, David Lillicrap MD Department of Pathology and Molecular Medicine, Richardson Laboratory, Queen's University, Kingston, ON, CanadaSearch for more papers by this authorRobert R. Montgomery MD, Robert R. Montgomery MD Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI, USA Blood Research Institute, Blood Center of Wisconsin, Milwaukee, WI, USASearch for more papers by this author First published: 21 March 2011 https://doi.org/10.1002/9781444329926.ch20Citations: 2 AboutPDFPDF ToolsRequest permissionExport citationAdd to favoritesTrack citation ShareShareShare a linkShare onEmailFacebookTwitterLinkedInRedditWechat Summary von Willebrand disease (VWD) is an attractive target disease for gene therapy. Both ex vivo and direct in vivo gene therapy approaches are being developed based on viral or non-viral gene delivery vectors. Since endothelial cells and megakaryocytes and their platelet progeny normally express von Willebrand factor (VWF), they constitute logical targets for gene therapy of VWD. Indeed, gene transfer into endothelial cells from dogs suffering from type 3 VWD using lentiviral vectors can result in de novo expression of adequately processed, fully functional VWF. However, even ectopic VWF expression in hepatocytes results in functional VWF that can correct the bleeding diathesis in VWF-deficient VWD mouse models. Despite this progress and early proof-of-concept studies, there is still a need to develop robust and safe gene therapy approaches for VWD, which would need to be validated in large animal models before moving forward with human clinical trials. References Sadler JE, Mannucci PM, Berntorp E, et al. Impact, diagnosis and treatment of von Willebrand disease. Thromb Haemost 2000; 84: 160–74. 10.1055/s-0037-1613992 CASPubMedWeb of Science®Google Scholar Ruggeri ZM. Von Willebrand factor. 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