Alternative trafficking of Weibel-Palade body proteins in CRISPR/Cas9-engineered von Willebrand factor-deficient blood outgrowth endothelial cells.

ABSTRACT:

Background

Synthesis of the hemostatic protein von Willebrand factor (VWF) drives formation of endothelial storage organelles called Weibel-Palade bodies (WPBs). In the absence of VWF, angiogenic and inflammatory mediators that are costored in WPBs are subject to alternative trafficking routes. In patients with von Willebrand disease (VWD), partial or complete absence of VWF/WPBs may lead to additional bleeding complications, such as angiodysplasia. Studies addressing the role of VWF using VWD patient-derived blood outgrowth endothelial cells (BOECs) have reported conflicting results due to the intrinsic heterogeneity of patient-derived BOECs.

Objective

To generate a VWF-deficient endothelial cell model using clustered regularly interspaced short palindromic repeats (CRISPR)

SUBMITTER: Schillemans M 

PROVIDER: S-EPMC6782018 | biostudies-literature | 2019 Oct

REPOSITORIES: biostudies-literature