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Ex vivo Improvement of a von Willebrand Disease Type 2A Phenotype Using an Allele-Specific Small-Interfering RNA

Von Willebrand disease (VWD) is the most common inherited bleeding disorder and is mainly caused by dominant-negative mutations in the multimeric protein von Willebrand factor (VWF). These mutations may either result in quantitative or qualitative defects in VWF. VWF is an endothelial protein that i...

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Detalhes bibliográficos
Publicado no:Thromb Haemost
Main Authors: de Jong, Annika, Dirven, Richard J., Boender, Johan, Atiq, Ferdows, Anvar, Seyed Yahya, Leebeek, Frank W. G., van Vlijmen, Bart J. M., Eikenboom, Jeroen
Formato: Artigo
Idioma:Inglês
Publicado em: Georg Thieme Verlag KG 2020
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7649061/
https://ncbi.nlm.nih.gov/pubmed/32803740
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1055/s-0040-1715442
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