公开(公告)号：EP3848039A1

公开(公告)日：2021-07-14

申请号：EP19869492.9

申请日：2019-08-30

Applicant: Korea Advanced Institute of Science and Technology

Inventor： HAN, Yong Mahn ,  DO, Hyo Sang

IPC: A61K31/7088 ,  A61K31/4439 ,  A61K45/06 ,  A61P43/00 ,  A23L33/10 ,  A23L33/13 ,  G01N33/68

Abstract: The present invention relates to a pharmaceutical composition for preventing or treating Fabry disease, containing a TSP1 protein inhibitor as an active ingredient. Particularly, in vascular endothelial cells produced by knocking out a TSP1 gene in induced pluripotent stem cells derived from a Fabry disease patient, of the present invention, the recovery of cell morphology, a decrease in the expression of a TSP1 gene, a decrease in the expression levels of a TSP1 protein and a phosphorylated-SMAD protein, which are anti-angiogenic factors, and an increase in the expression levels of a KDR protein and an eNOS protein, which are angiogenic factors, have been confirmed, and thus a TSP1 gene expression inhibitor or a TSP1 protein activity inhibitor can be effectively used in the treatment of Fabry disease.