Yvonne Jongejan: Allele-Selective Silencing Therapy for VWD and Thrombotic Disorders
Yvonne Jongejan, Postdoctoral researcher at Leiden University Medical Center, reposted from RPTH Journal on LinkedIn:
”A nice overview in RPTH of the work Annika de Jong, Noa Linthorst, Isabel Bär, Jeroen Eikenboom and myself have done and are still doing to develop an allele-selective silencing therapy for von Willebrand disease and thrombotic disorders.”
Quoting RPTH Journal‘s post about a recent article by Jeroen Eikenboom et al:
”What if the future of VWF therapy is not replacing the protein, but silencing the wrong allele?
This RPTH State-of-the-Art review highlights allele-selective VWF silencing: a precision siRNA strategy designed to switch off only one VWF allele.
For dominant-negative VWD, this could remove mutant VWF and restore healthier multimer formation. For thrombotic disorders linked to high VWF, it may offer a controlled way to lower VWF without pushing patients into bleeding risk.
One allele. Two clinical directions. A new precision-medicine angle for VWF biology.”
Title: Allele-selective von Willebrand factor silencing
Authors: Jeroen Eikenboom, Noa Linthorst, Yvonne Jongejan
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