Year: 2015
Type: conference-paper
Abstract: Fibrodysplasia Ossificans Progressiva (FOP) is a rare monogenetic disorder in which patients develop heterotopic ossification (HO). A heterozygous mutation in BMP type I receptor ALK2 results in hyper-sensitized BMP signaling. The aim of this study is to identify small molecules which can selectively inhibit this overactive BMP pathway. Thirteen FDA-approved small molecules were tested on their effect on BMP6-induced target gene expression, alkaline phosphatase activity and mineralization in KS483 cells. We identified cryptotanshinone as a small molecule able to inhibit BMP signaling. In conclusion, cryptotanshinone could be a novel small molecule inhibitor of the overactive BMP signaling pathway in FOP.
Institution: Leiden University
Language: English
Subfield: Rheumatology
Field: Medicine
Domain: Health Sciences
Open Access status: green
OpenAlex ID: W2249648378