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Amelioration of a von Willebrand disease type 2B phenotype in vivo upon treatment with allele-selective siRNAs

16 Jan 2025

Abstract excerpt

ABSTRACT: Treatment options for the bleeding disorder von Willebrand disease type 2B (VWD2B) are insufficient and fail to address the negative effects of circulating mutant von Willebrand factor (VWF). The dominant-negative nature of VWD2B makes functionally defective VWF an interesting therapeutic target. Previous in vitro studies have demonstrated the feasibility of allele-selective silencing of mutant VWF...

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PMID: 39820471
DOI: 10.1182/bloodadvances.2024014601

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