PHARMACEUTICAL COMPOSITION FOR PREVENTING OR TREATING FABRY DISEASE, CONTAINING TSP1 PROTEIN INHIBITOR AS ACTIVE INGREDIENT

    公开(公告)号:US20220034908A1

    公开(公告)日:2022-02-03

    申请号:US17280567

    申请日:2019-08-30

    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.

Patent Agency Ranking