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Allele-selective von Willebrand factor silencing | Synapse
March 3, 2026
Open Access
Allele-selective von Willebrand factor silencing
RA
Ryanne A. Arisz
Erasmus MC
NL
Noa A. Linthorst
Leiden University Medical Center
YJ
Yvonne K. Jongejan
Leiden University Medical Center
Puntos clave
Allele-selective silencing led to a significant decrease in von Willebrand factor levels.
Specifically, a 70% reduction in vWF expression was observed in treated cells.
Analysis of gene expression using bioengineering techniques revealed the success of targeted interventions.
These findings may enable more effective hemophilia treatments through genetic manipulation.
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Arisz et al. (Thu,) studied this question.
synapsesocial.com/papers/69a75bb6c6e9836116a2389d
https://doi.org/https://doi.org/10.1016/j.rpth.2026.103366
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