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Article

High and long-term von Willebrand factor expression after Sleeping Beauty transposon-mediated gene therapy in a mouse model of severe von Willebrand disease.

Journal of thrombosis and haemostasis : JTH - 1 Mar 2018

Portier I, Vanhoorelbeke K, Verhenne S, Pareyn I, Vandeputte N, Deckmyn H, Goldenberg D S, Samal H B, Singh M, Ivics Z, Izsvák Z, De Meyer S F

Abstract excerpt

Essentials von Willebrand disease (VWD) is the most common inherited bleeding disorder. Gene therapy for VWD offers long-term therapy for VWD patients. Transposons efficiently integrate the large von Willebrand factor (VWF) cDNA in mice. Liver-directed transposons support sustained VWF expression with suboptimal multimerization. SUMMARY: Background Type 3 von Willebrand disease (VWD) is characterized by complete...

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PMID: 29288565
DOI: 10.1111/jth.13938

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VWF

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