Amelioration of a von Willebrand disease type 2B phenotype in vivo upon treatment with allele-selective siRNAs.
Linthorst NA, Jongejan YK, Dirven RJ, Laan SNJ, Bierings R, Casari C, Cordfunke RA, Dahlman JE, Dolezal N, Drijfhout JW, Leebeek FWG, Ruhaak LR, Schrader Echeverri E, Voorberg J, van Vlijmen BJM, Denis CV, Eikenboom JCJ.
Linthorst NA, et al. Among authors: ruhaak lr.
Blood Adv. 2025 Jan 28;9(2):310-320. doi: 10.1182/bloodadvances.2024014601.
Blood Adv. 2025.
PMID: 39820471
Free PMC article.