Composition for preventing or treating cardiofaciocutaneous syndromeCFC 신드롬의 예방 또는 치료용 조성물

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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.
Assignee
KAIST
Country
US (United States)
Application Date
2019-02-07
Application Number
16269984
Registration Date
2021-07-13
Registration Number
11059875
URI
http://hdl.handle.net/10203/286847
Appears in Collection
BS-Patent(특허)
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