公开(公告)号：US20240376437A1

公开(公告)日：2024-11-14

申请号：US18444289

申请日：2024-02-16

Applicant: University of Washington

Inventor： Hongxia Fu ,  Benjamin Freedman ,  Jose A. Lopez

IPC: C12N5/071 ,  C12M3/00 ,  C12M3/06 ,  C12N9/22 ,  G01N33/50

Abstract: The present disclosure provides methods and compositions useful for generating cellular models of VWF disease from human pluripotent stem cells. In one aspect, the present disclosure relates to a method for the generation of an in vitro cellular model of VWF disease comprising VWF disease-relevant endothelial cells derived from human pluripotent stem cells (hPSCs). In an embodiment, the human pluripotent stem cells (hPSCs) are treated with an agent effective in suppressing or deleting at least one gene sequence encoding a protein and/or a subunit thereof, where the protein and/or subunit thereof is associated with VWF-linked secretion. The present disclosure also provides uses of the cellular models disclosed herein.