公开(公告)号：US20210284705A1

公开(公告)日：2021-09-16

申请号：US17331036

申请日：2021-05-26

Applicant: Korea Advanced Institute of Science and Technology

Inventor： Yong Mahn Han ,  Jung Yun Choi ,  Bum Soo Kim

IPC: C07K14/51 ,  A61K31/7088 ,  C12Q1/02 ,  A61K31/519 ,  A61K31/5377 ,  A61K31/4439 ,  A61K31/4709 ,  A61P9/00 ,  G01N33/50

Abstract: The present invention relates to a pharmaceutical composition for the prevention or treatment of CFC (cardiofaciocutaneous) syndrome comprising a TGF-β signaling pathway inhibitor or a BMP signaling pathway activator. The treatment of SB-431542 or BMP4 protein was confirmed to increase the ALP activity and bone mineral deposition in the osteoblasts originated from the induced pluripotent stem cells derived from CFC syndrome patients. Thus, the TGF-β signaling pathway inhibitor containing SB-431542 or the BMP signaling pathway activator containing BMP4 protein can be effectively used for the prevention or treatment of CFC syndrome.

公开(公告)号：US20190241635A1

公开(公告)日：2019-08-08

申请号：US16269984

申请日：2019-02-07

Applicant: Korea Advanced Institute of Science and Technology

Inventor： Yong Mahn Han ,  Jung Yun Choi

IPC: C07K14/51 ,  A61K31/7088 ,  C12Q1/02 ,  A61P9/00 ,  A61K31/5377 ,  A61K31/4439 ,  A61K31/4709 ,  A61K31/519

CPC classification number: C07K14/51 ,  A61K31/4439 ,  A61K31/4709 ,  A61K31/519 ,  A61K31/5377 ,  A61K31/7088 ,  A61P9/00 ,  C12Q1/025

Abstract: The present invention relates to a pharmaceutical composition for the prevention or treatment of CFC (cardiofaciocutaneous) syndrome comprising a TGF-β signaling pathway inhibitor or a BMP signaling pathway activator. The treatment of SB-431542 or BMP4 protein was confirmed to increase the ALP activity and bone mineral deposition in the osteoblasts originated from the induced pluripotent stem cells derived from CFC syndrome patients. Thus, the TGF-β signaling pathway inhibitor containing SB-431542 or the BMP signaling pathway activator containing BMP4 protein can be effectively used for the prevention or treatment of CFC syndrome.

公开(公告)号：US11059875B2

公开(公告)日：2021-07-13

申请号：US16269984

申请日：2019-02-07

Applicant: Korea Advanced Institute of Science and Technology

Inventor： Yong Mahn Han ,  Jung Yun Choi ,  Bum Soo Kim

IPC: C07K14/51 ,  A61K31/7088 ,  A61K31/5377 ,  A61K31/4439 ,  A61K31/4709 ,  A61K31/519 ,  A61P9/00 ,  C12Q1/02 ,  G01N33/50

Abstract: The present invention relates to a pharmaceutical composition for the prevention or treatment of CFC (cardiofaciocutaneous) syndrome comprising a TGF-β signaling pathway inhibitor or a BMP signaling pathway activator. The treatment of SB-431542 or BMP4 protein was confirmed to increase the ALP activity and bone mineral deposition in the osteoblasts originated from the induced pluripotent stem cells derived from CFC syndrome patients. Thus, the TGF-β signaling pathway inhibitor containing SB-431542 or the BMP signaling pathway activator containing BMP4 protein can be effectively used for the prevention or treatment of CFC syndrome.