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Journal Articles

Allele-selective Disruption of Pathogenic VWF Variants in Type 2 Von Willebrand Disease using CRISPR/Cas9

Isabel Bär, Stijn A. Groten, Alastair Barraclough, Petra E. Bürgisser, Calvin van Kwawegen, Peter J Lenting, Iris van Moort, Jeroen C.J. Eikenboom, Frank W.G. Leebeek, Jan Voorberg, Maartje van den Biggelaar, Ruben Bierings

Blood Adv bloodadvances.2025018760.

https://doi.org/10.1182/bloodadvances.2025018760

Published: 2025

Includes: Supplemental data

Journal Articles

Amelioration of a von Willebrand disease type 2B phenotype in vivo upon treatment with allele-selective siRNAs

Noa A. Linthorst, Yvonne K. Jongejan, Richard J. Dirven, Sebastiaan N. J. Laan, Ruben Bierings, Caterina Casari, Robert A. Cordfunke, James E. Dahlman, Natasja Dolezal, Jan Wouter Drijfhout, Frank W. G. Leebeek, L. Renee Ruhaak, Elisa Schrader Echeverri, Jan Voorberg, Bart J. M. van Vlijmen, Cécile V. Denis, Jeroen C. J. Eikenboom

Blood Adv (2025) 9 (2): 310–320.

https://doi.org/10.1182/bloodadvances.2024014601

Published: 2025

Includes: Supplemental data

Journal Articles

O-glycan determinants regulate VWF trafficking to Weibel-Palade bodies

Ellie Karampini, Dearbhla Doherty, Petra E. Bürgisser, Massimiliano Garre, Ingmar Schoen, Stephanie Elliott, Ruben Bierings, James S. O’Donnell

Blood Adv (2024) 8 (12): 3254–3266.

https://doi.org/10.1182/bloodadvances.2023012499

Published: 2024

Journal Articles

GDP/GTP exchange factor MADD drives activation and recruitment of secretory Rab GTPases to Weibel-Palade bodies

Marije Kat, Petra E. Bürgisser, Hans Janssen, Iris M. De Cuyper, Ianina L. Conte, Alistair N. Hume, Tom Carter, Jan Voorberg, Coert Margadant, Ruben Bierings

Blood Adv (2021) 5 (23): 5116–5127.

https://doi.org/10.1182/bloodadvances.2021004827

Published: 2021

Includes: Supplemental data