Correction of a dominant-negative von Willebrand factor multimerization defect by small interfering RNAs-mediated allele-specific inhibition of mutant von Willebrand factor.

CONCLUSIONS: Allele-specific siRNAs are able to distinguish VWF alleles based on one nucleotide variation and are able to improve a severe multimerization defect caused by VWF p.Cys2773Ser. This holds promise for the therapeutic application of allele-specific siRNAs in dominant-negative VWD. This article is protected by copyright. All rights reserved. PMID: 29734512 [PubMed - as supplied by publisher]

https://www.ncbi.nlm.nih.gov/pubmed/29734512?dopt=Abstract

Source: Thrombosis and Haemostasis - May 7, 2018 Category: Hematology Authors: de Jong A, Dirven RJ, Oud JA, Tio D, van Vlijmen BJM, Eikenboom J Tags: J Thromb Haemost Source Type: research