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Article

In vivo modulation of a dominant-negative variant in mouse models of von Willebrand disease type 2A.

Journal of thrombosis and haemostasis : JTH - 1 Jan 2021

Campioni Matteo, Legendre Paulette, Loubiere Cécile, Lunghi Barbara, Pinotti Mirko, Christophe Olivier D, Lenting Peter J, Denis Cécile V, Bernardi Francesco, Casari Caterina

Abstract excerpt

Essentials Treatment options for von Willebrand disease (VWD) patients are limited. The p.P1127_C1948delinsR deletion/variant is a useful model to study VWD in vitro and in vivo. Counteracting dominant-negative effects restores von Willebrand factor multimerization in mice. This is the first siRNA-based treatment applied to a mouse model of VWD-type 2A. ABSTRACT: Background Treatment options for patients...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 33047469
DOI: 10.1111/jth.15131

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