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Correcting dominant-negative von Willebrand disease
In this issue of the Journal of Thrombosis and Haemostasis, Campioni et al report correction of dominant-negative von Willebrand disease (VWD) in vivo for the first time. In particular, they demonstrate that targeted inhibition of a mutant VWF allele can significantly improve bleeding phenotype in a Type 2A VWD murine model. Collectively, these data move us another step closer to developing personalized approaches for the treatment of VWD patients that extend beyond traditional von Willebrand factor (VWF) infusion therapy.
NIH for the Zimmerman Program (HL081588)
Science Foundation Ireland Principal Investigator Award (11/PI/1066)
Health Research Board Investigator Lead Project Award (ILP-POR-2017-008)
National Children's Research Centre Project Award (C/18/1)
CommentsThis is the peer reviewed version of the following article, Karampini E, O'Donnell JS. Correcting dominant-negative von Willebrand disease. Journal of Thrombosis and Haemostasis. 2021;19(1):55-57, which has been published in final form at https://doi.org/10.1111/jth.15123. This article may be used for non-commercial purposes in accordance with Wiley Terms and Conditions for Self-Archiving.
Published CitationKarampini E, O'Donnell JS. Correcting dominant-negative von Willebrand disease. Journal of Thrombosis and Haemostasis. 2021;19(1):55-57.
Publication Date6 January 2021
- Irish Centre for Vascular Biology
- School of Pharmacy and Biomolecular Sciences
- Vascular Biology
- Accepted Version (Postprint)