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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: Georg Thieme Verlag KG 2020
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7649061/
https://www.ncbi.nlm.nih.gov/pubmed/32803740
http://dx.doi.org/10.1055/s-0040-1715442

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7649061/
https://www.ncbi.nlm.nih.gov/pubmed/32803740
http://dx.doi.org/10.1055/s-0040-1715442

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